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Retinal Researchers May Be Looking for You

A patient registers with My Retina Tracker.One of the biggest challenges in overcoming rare retinal diseases is, well, that they’re rare. There’s limited information about the conditions in humans, making it difficult for researchers to understand why they cause blindness and develop vision-saving treatments.

Collecting retinal-disease data, and making it available to scientists, is one of the major driving forces behind the development of My Retina Tracker, the Foundation’s free, secure and confidential registry for people with inherited retinal diseases. One thing I do want to make clear—the data collected is protected and only shared, with each patient’s consent, for legitimate research purposes. Only disease-related data can be accessed by researchers; no personal information is shared. FFB staff will notify registrants if they match a researcher’s clinical-trial criteria.

When the Foundation launched My Retina Tracker in 2014, we knew investigators and companies would want the data provided by patients and families. And even though we’re still in a growth phase in terms of registration, it’s remarkable how many companies and investigators are already asking for disease information.

In our first year of operation alone, we’ve had data inquiries from more than eight companies and applications for data access from 10 individual researchers. Many of the companies are interested in identifying people who may be eligible for participation in clinical studies launching soon. They’re also looking at patient and disease numbers to determine if future therapy development efforts and clinical trials are warranted.

For example, Applied Genetic Technologies Corporation, a developer of retinal-disease gene therapies asked us to query My Retina Tracker to identify potential candidates for its gene-therapy clinical trial for X-linked retinoschisis. The company also intends to initiate studies for achromatopsia (day blindness) and X-linked retinitis pigmentosa in the not-too-distant future.

Looking forward, both retinal-disease patients and FFB still have a lot of work ahead to populate My Retina Tracker; we need thousands of more registrants. FFB’s job is to get the word out.

But, ultimately, it is up to the affected individuals and families to register and provide as much current disease information as possible. While genetic and vision test results are valuable, any information from patients and their blood-related family members (both affected and unaffected) is helpful. Registrants can always sign up and add more information later, as it becomes available.

Please send an e-mail to coordinator@myretinatracker.org if you have any questions or need registration assistance.

Thanks for helping drive sight-saving research.


69 Responses to 'Retinal Researchers May Be Looking for You'

  1. Luka Tovornik says:

    I was diagnosed with retinitis pigmentosa this year, it’s heredetary because my mother has it,a and her my we belive that my grandfather had it.I aplaude your efforts,and wish you to find a cure for all of us.

    • Burak says:

      Hello, I’m a 19-year old boy that waiting to cure my eyes for 6 years. I have the disease of Stargardt. I hope this is my luckly year. Can you contact me if you can help me. Thank you.

      • EyeOnTheCure says:

        You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
        http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

        You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

        Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

        Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

        Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

        Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
        *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

        Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

        Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

        To read more about the partnership between Vision Medicine and FFB, see the following web link:
        http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  2. roxanne griffin says:

    where do we register? have RP from mother/grandmother

  3. My son suffered from retina pigmentosa assosiated with lawerence moon bedil syndrom . My son age has 8 years old and weight 38 kgs . please help the my son , Please information about the R.P treatmet and this type of above Syndrom. Please tell me about the above information.

  4. Lisa wilkerson says:

    I am excited that there are sites encouraging people thst has been Dx. With retinal disease.I was diagnosed with Stargerdts disease approx 3 years ago and still waiting to hear that a cure is found.

  5. CHARLES MCMULLEN says:

    HELLO I AM A 64 YEAR YOUNG MAN —I HAVE BEEN LEGALLY BLIND FROM THE AGE OF 14 —AT LEAST THIS IS WHEN I WAS
    diagnosed
    WITH —AT THE TINE IT WAS CALLED
    juvenile MACULAR DEGENERATION —–NOW KNOWN AS –STARGARDTS—I DON’T KNOW WHAT ELSE I CAN ADD TO THIS —YOU HAVE MY EMAIL —CONTACT ME IF YOU NEED OR WANT TO —THANKS CHUCK -MCMULLEN PGH PA
    PS MY BROTHER —8 YEARS YOUNGER THAN ME –ALSO WAS AFFECTED —BUT NOT UNTIL HIS MID FORTY’S

  6. Linda Monds says:

    I have Usher Syndrome. RP and deafness. Seeing yellow/orange colors and basically only seeing outlines of things and no details. I am 47 years old and the past 8’months it seems to have moved much quicker.

  7. gilbert m says:

    thanks for your work.Is it posiible for people in eastafrica to undergo tests!

  8. Andrew Monk says:

    My father is 70yrs old and fit as a fiddle. He has had Stargerts since he was 13. In many ways he accepts nothing can be done to help the actual condition itself but always talks of wishing he had his sight back.

    Please get in touch if you think you can help.

    Kind regards

    Andrew Monk

    • EyeOnTheCure says:

      Your father will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  9. Sarah says:

    My 2 year old son has Alstrom Syndrome and severe photophobia from cone cell dystrophy. Please continue with gene therapy research to save his sight.

  10. Andra Loy Carson says:

    My son is 34 and was diagnosed with Glaucoma and Retinal Pigmentosa within the last 2 years and is expetiencing changes NOW . He was stable for the past ten months but three days ago experienced more loss.

    I would like him to be able to talk to other young people experiencing the same issues. He has 15% vision in one eye and 85,% in the other and is right now ( appt on Tues the 5th) considering, not yet doing a TRABULECTOMY on his good eye. I have one friend age 62 who had the trabulectomy and said having done it he wishes he hadn’t.
    We need information from other people who have had a TRABULECTOMY. Please contact me at:

    loycarson@gmail.com

    If there is anyone who can talk to me I need co.tacts, information. We are in Sa. Francisco, Ca. My son is isolating himself but if there is another person he can talk to I believe he will.

    Thanx

    • Steven Tucker says:

      My name is Steve Tucker a 57 year old male and a father of 3 kids.I currently have some usable vision and still work.I have dealt with night blindness my entire life ,and understand the daily struggles your son is dealing with.The constant daily worries of the blindness to come.I live in Novato CA and work in San Fransisco.I to am trying to find all clinical trials and have registered on many waiting lists.No calls back but there are at least 2 ongoing gene therapy clinical trials at UC Irvine in LA.There is Dr Birch at Southwest Eye Center in Texas. They are currently many other phase 1 and going into phase 2 trials.All are for RP patients with advanced vision loss. Please dont give up hope because they have told me the patients are responding very well.Like many others i wait for my angel of sight to help me one day.If I can help in any way please let me know.

  11. Fiona bert says:

    I was diagnosed with stargrdts in 2012 would love to register really finding life difficult

    • EyeOnTheCure says:

      You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  12. nawazgul says:

    my age 23 years old and brother and uncle retinitis pigmentosa problem please help me contacts me 9230383593217

    • EyeOnTheCure says:

      You and your brother should consider genetic testing to try and identify the mutant gene responsible for causing the RP. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, you both should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  13. I have r p and was wondering if there is a cure

  14. Lone says:

    So my gf of a year recently was diagnosed with RP and she’s been in and out between having kids when we get married because she’s afraid that they’ll hate her for “giving” them RP, which i talk sense into her afterwards. I just really hope a cure gets found soon, I hate seeing her be so distraught and I’d gladly donate to find the cure in future years.

    • EyeOnTheCure says:

      If your girlfriend has recessive RP, the most common form of RP, your children will not have RP but will be carriers. You should know that there are three inherited forms of RP: recessive, dominant and X-linked. For information on inheritance types, please see the following web link to download a PDF document on inheritance:
      http://www.blindness.org/sites/default/files/inheritance_of_retinal_degeneration_-_july_2012.compressed.pdf

      Your girl friend should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, your girl friend should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

    • Steven Tucker says:

      First of all Im sorry for your gf eye disease.I to have 3 kids who do nont have my Retinitis Pigmentosa.There are certain genes passed on from your parents.Some are recessive and others dominant.An opthimologist can direct you some but you can get genetic tests to determine how genetically your kids to be would be passed on.Hope this helps some. Good luck

  15. Jordin McEntire says:

    I have recently been diagnosed with RP and I am only 24. This is very scary to me as I have seen my vision change quickly in the past couple of months. I was wondering if anyone was diagnosed with this where they do not loose all their vision. Again I am so young and I do not want to go blind and with there being no cure I am really nervouse. But I am walking with faith at this point not with sight!

    • EyeOnTheCure says:

      You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

    • Sara says:

      Just want to let you know that my mother of 45 years of age has it and she still sees, there is no complete loss of vision, just a decline.

    • Andy says:

      Hey Jordan,
      I’m 53 now and was diagnosed w RP (more specifically Ushers Syndrome type 2) around 24 yrs old too. While I’ve had both significant peripheral and night vision degredation since my late teens and now have roughly 20 degrees visual field. I still live life, work, provide for family… My future is completely by faith, not always understanding the whys but knowing the Lord is good and His purpose involves more than just me.

      Andy

    • carol atkinson says:

      Don’t give in to fear. You are young, but also a lot older than many people diagnosed with RP. I was 42, at the peak of my career and a single mother of 4 when I was diagnosed. I am legally blind. It did really change my life….but it never went to total blindness. Turn over every leaf, join every support group you can find….and believe in yourself.

    • alejandro rodriguez says:

      Hi, I am 48 and I was diagnosed with RP on Oct 2014. I have lost almost all my peripheral vision but other than that I am fine. My mom had also RP and she never lost her sight completely and she was 78 when she passed.

      Every person is different but I hope my answer helps.

  16. Mary Gray says:

    My husband has RP, diagnosed just as he turned 30. Both of his uncles on his mother’s side were affected also. No female relatives affected, and his two sons are unaffected. We are thinking this looks like a genetic condition linked to the female chromosome. If there are any trials to affect this condition, he would be interested.he is now 72..he still has a little vision, but not much.

  17. Judy Gibney says:

    My friend Patrick has been suffering with rp as well as her older sister. We live in the Austin, Texas area and we need information on active studies or treatments in this area. Please help.

    Judy Gibney

    • EyeOnTheCure says:

      Your friend should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether Patrick’s disease gene is identified or not, he should still consider enrolling in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  18. Karen Lamb says:

    Hi my daughter was diagnosed with stargardts disease 4 years ago, she will be 21 this summer. One year ago this coming Tuesday 16th February, we will celebrate her daughters 1st birthday. My daughter, partner and granddaughter will move into their new place sometime in March. I would love nothing more than something being done for my daughters sight. It’s heartbreaking as we can see her eyesight has deteriorated. I wish I was a huge lottery winner to pay for help for her.

    • EyeOnTheCure says:

      You and your daughter will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also ask your daughter to enroll in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  19. David cruz says:

    My mother was just diagnosed with retinitis pigmentosa,as a son I am devastated. All I can do is research and look for a cure or treatment for this. There should be or there should be one out soon,if they got procedures to change people’s eye color they should have procedures for this. All I can do is pray to a higher power for a cure or treatment to be out there or to come out soon.

  20. Joy says:

    My friend is blind because of Congenital glaucoma. Her right eye was removed in 2001. She is interested in this research. Please contact me if she is needed for your research.

  21. Vanessa Hayward says:

    I was diagnosed with Stargardts at the age of 28. I am now 36 and can feel my central vision worsening. At this point, I am still able to work and drive. I am desperately hoping to find a way to slow (stop) the progression. I am a high school teacher and a mother to two young boys. Any information would be strongly appreciated!

    • EyeOnTheCure says:

      Hi Vanessa, You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  22. Nour Saliba says:

    My son is 11 years old and diagnosed with stargardts disease 6 mounth ago.It is very hard for him to accept this.
    Please contact us for your research.

    • EyeOnTheCure says:

      Dear Nour, You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  23. Steph says:

    My bf was diagnosed with RP about 2 yrs ago @ age 36. He has seen progression in vision loss over the last 6 mths. No one in his family has ever been diagnosed with this condition, so to hear most everyone state that it is genetic is concerning. Praying for a cure, but hoping for a miracle. Any studies or information for said studies would be great. My prayers go out to everyone with visual impairment.

    • EyeOnTheCure says:

      Your boy friend should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  24. Rasel says:

    I heard about this treatment going on in Canada by the China Dr, My brothers has RP and he is 33 years old now. So we plan to go there for getting his treatment. I don’t know what actually will happen. China Dr. will treat us by some China medicine with his therapy. If anyone have any idea about that China Dr, could you guys help us to let us know more about this Dr? We plan to go next month.

  25. Rosa Huertas says:

    My son 32 was diagnosed with vitilliforum disorder and is progressing . We being told isn’t a cure. Is there any studies or information?

    • EyeOnTheCure says:

      Vitelliform macular dystrophy or vitelliform dystrophy is a genetic eye disorder that can cause progressive vision loss. This disorder affects the retina, specifically cells in a small area near the center of the retina called the macula. Mutations in one of several genes can cause vitelliform dystrophy. You should consider genetic testing to try and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, one may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:
      http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  26. Garry Schaffer says:

    I have had recent retina surgery in my right eye to reattach a torn retina. I now have only 20/70 visionm there. Are there any companies doing research for this in Dallas, Texas?

  27. Maureen W says:

    My son, now 22, was diagnosed with RP at 16 years old. My older brother also has it. He was diagnosed at the same age as my son, but went blind at 30. They have been doing ‘studies’, it seems like forever, but now, actual clinical trials. My son and myself are very interested in stem cell therapy or retinal transplant. I’ve heard these trials have started in the Phillapenes and moving into California. We live on the east coast, but are willing to travel anywhere in the U.S. He is also affected with Aspeger’s Syndrome, in the Autism spectrum, it would be so helpful if he could get some sight back. He has started to isolate himself and he is way too young to go that route. Please contact.

  28. Gail Osmun says:

    Please, my son has ROP. Sight in right eye can be corrected to 20/60. Vision in left eye is limited to light/dark due to retinal hemorrhage at 6 weeks of age along with retinal detachment. Is there anything that be done to help him?

  29. I am a (almost) 43 years old and I was diagnosed with Stargardt’s Disease in 2007. I am seeking whatever clinical trials I may qualify for to find a way to reduce it’s progression or possibly find a cure. Please contact with me with any information that may be helpful in this endeavor. I look forward to a response. Thank you for your consideration.

    • EyeOnTheCure says:

      You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 In order to participate in this or any other gene therapy trial, you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      You should also consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Finally, below is a list of pharmaceutical companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) completed Phase 3 trials for dry AMD in June 2016. Unfortunately, the drug failed to slow lesion growth in over 500 patients with geographic atrophy, a form of dry AMD. Because of a strong demonstration of efficacy in the Stargardt mouse model, Acucela is considering initiating a study to explore potential benefits of Emixustat in patients with Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660

      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future.

      To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  30. Tina Omerović says:

    Hi.

    I am 23 years old and last year I was diagnosed with APMPPE. It is extremely rare and unresearched. My central vision is disappearing. Doctors said that some of my photoreceptors are sick or dead. I heard about retinal stem cell therapy.
    Are you familiar with APMPPE or with mentioned therapy.
    Is there anything that could help me with my problem?

    Kind regards,
    Tina

    • EyeOnTheCure says:

      Unfortunately, Acute posterior multifocal placoid pigment epitheliopathy is not one of the diseases that is being researched by FFB scientists. However, you may want to consider contacting the National Eye Institute that funds a broader range of eye diseases. Here is the link:
      https://nei.nih.gov/

  31. Pauline says:

    My wife was diagnised with RP in her late 20’s. She is now 48.Has no nihht vision but still good day vision. Her dad and brother also affected. Would like to contribute.

  32. Adele says:

    When I was 23 I was diagnosed with RP, but suspect that I have Usher Syndrome. Not yet confirmed. It was said that I’ve become hard hearing as a baby due to high fever. I’m now 41 years old, suffering from night blindness, tunnel vision and my details is disappearing. So far I can track no family history of either RP nor Usher Syndrome

    • EyeOnTheCure says:

      Dear Adele, It is possible that you have Usher syndrome of which there are three general types. People with Usher syndrome type 1 (USH1) are born completely deaf and experience problems with balance. The first signs of RP — night blindness and loss of peripheral vision — usually appear in early adolescence. In Usher syndrome type 2 (USH2), newborns have moderate to severe hearing impairment and the RP symptoms typically start shortly after adolescence. Visual problems progress less rapidly than in Usher type 1 and hearing loss usually remains stable. A rarer third type of Usher syndrome (USH3) was documented in 1995. Children with USH3 are usually born with good or only mild impairment of hearing. Their hearing and vision loss is progressive, starting around puberty. Balance may be affected. For more detailed information on Usher syndrome, please see the following web link: http://www.blindness.org/eye-conditions/usher-syndrome

      Genetic testing is the best way to confirm that you have Usher syndrome and identify the mutant gene responsible for causing the disease. If the gene is identified, medical databases such as PubMed (http://www.ncbi.nlm.nih.gov/pubmed/) can be searched to identify any research that is being conducted. With a molecular diagnosis, you may qualify for one of the gene therapy trials that are taking place. The Foundation Fighting Blindness is currently supporting a gene therapy trial for Usher type 1B. For more information on this clinical trial, please see the following link: http://www.blindness.org/foundation-news/usher-syndrome-gene-therapy-study-begins-oregon

      To participate in any gene therapy trial, you must first positively identify the gene mutation. For more information on genetic testing, please see the following link:
      http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf

      Whether your disease gene is identified or not, you should still consider enrolling in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

      You may also find it helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. Thank you for your support that is helping to accelerate the development of new safe and effective treatments for inherited retinal disease.

  33. Roger Wjitcomb says:

    I am 80. Is age related MACD one of your areas of interest? If so. I would like to share information.

  34. Jay says:

    I am 40 year old male diagonised with Central Serous Retinoparhy (CSR) and most of my light cells are damaged.
    I am interested in this research. Could you please contact me do that I can share all my medical record with you .

  35. Kristen j says:

    My 60 yr old father had sudden vision loss and was diagnosed with autoimmune optic neuropathy. Would his situation apply?

  36. Mark says:

    I AM MARK. FOR THE PAST 12 YEARS i HAVE BEEN DIAGNOSED WITH SICKLE RETINOPATHY. I AM INTERESTED TO FIND OUT IF THERE IS ANY TREATMENT, PARTICULARLY GENE THERAPY, THAT IS CURRENTLY AVAILABLE TO TREAT OR REVERSE THIS DISEASE.

    • EyeOnTheCure says:

      Hi Mark,

      FFB doesn’t fund research for sickle retinopathy. Some drugs for wet age-related macular degeneration may help people with sickle retinopathy, but you need to follow-up with a retinal specialist to determine if any treatment is warranted or available for you.

  37. Nancy Jean Korb says:

    I have plaquenil toxic maculopathy. Is there any treatment for this? My vision is tunneling. I am a 70 y.o. female. I was diagnosed with Sjogren’s in 1995 at age 48.

    • EyeOnTheCure says:

      FFB funds research for inherited (genetic) retinal diseases. The research we support does not apply to your condition. We suggest contacting an academic research center (perhaps in your area) that may do more research for corneal or retinal damage.

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