Spark Therapeutics’ vision-restoring RPE65 gene therapy has received marketing approval from the U.S. Food and Drug Administration, becoming the first gene therapy to gain regulatory approval in the U.S. for the eye or any inherited condition.
Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored vision in a clinical trial for people between the ages of 4 and 44 with Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. Study participants with severe vision loss reported putting away their navigational canes, seeing stars, being able to read, and recognizing faces of loved ones. Vision restoration has persisted for at least three years. The treatment is also designed to work for people with retinitis pigmentosa (RP) caused by RPE65 mutations.
On October 12, 2017, an FDA Advisory Committee voted 16 to 0 in recommending marketing approval for LUXTURNA.
The Foundation Fighting Blindness invested about $10 million in more than a decade of lab research that made possible the RPE65 gene therapy clinical trial at the Children’s Hospital of Philadelphia (CHOP). FFB-funded research included: studies to understand the role of RPE65 in vison and retinal disease, development of animal models with RPE65 mutations, RPE65 gene therapy testing and development, and support for early clinical research at CHOP.
“This is truly a historical moment in the fight against blindness. We are delighted by the FDA’s approval of LUXTURNA for people with RPE65 mutations,” says Stephen Rose, PhD, chief research officer at FFB. “LUXTURNA’s approval also provides affirmation that gene therapies can be a safe, effective, and commercially viable approach to treating many forms of blindness, providing a big boost to an already burgeoning field. The approval is great news for a broad spectrum of people with inherited retinal diseases.”
Retinal gene therapy clinical trials are underway for people with a wide range of retinal diseases including: X-linked RP, choroideremia, Usher syndrome type 1B, Stargardt disease, X-linked retinoschisis, and achromatopsia.
More than 10 million people in the U.S., and millions more around the world, have retinal degenerative diseases.
“We are thrilled for the patients whose lives will change because of this treatment,” says David Brint, Foundation Fighting Blindness chairman. “And we are also pleased to have this concrete example of the strength of the Foundation’s strategy of investing early in promising treatments in order to attract later industry investment that can usher the treatments through clinical trials and FDA approval.”
“LUXTURNA will be life changing for people with RP and LCA caused by RPE65 mutations. For them, the treatment may well mean the difference between relying on assistive technologies or other people and living a life of independence. Also important is the momentum this approval provides to other gene-based therapies — for the eye and other diseases — now in the pipeline,” says Benjamin Yerxa, PhD, Foundation chief executive officer.
The LUXTURNA gene therapy involves injection of healthy copies of RPE65 underneath the retina. The RPE65 copies are contained in a human-engineered virus — known as an adeno-associated virus or AAV — which is designed to readily penetrate retinal cells to deliver the therapeutic genetic cargo. A single treatment is expected to last several years.
Spark Therapeutics, which holds the biologics license for LUXTURNA and conducted the clinical trials that showed its safety and efficacy, will also manage the treatment rollout. Spark has announced that in order to ensure the treatment is safely administered, it will only be available through a small number of centers of clinical excellence around the country. Spark has also expressed its commitment to educate third-party payers about the value of LUXTURNA and to work to help ensure treatment access to all eligible patients.
Anyone in need of more information about LUXTURNA should contact Spark Therapeutics at 1-833-SPARK-PS (833-772-7577). Another resource for information is www.luxturna.com.
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. 
As the Foundation's Director, Science Communica- tions, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. 
My husband has RP. Is this current gene therapy (Luxterna) helpful for him at this time?
LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
This is wonderful news, Is there a program that my friend can take part in? He is 63 years old and has Retinitis pigamentosa. I certainly pray that he can participate in the program.
LUXTURNA is only for people with mutations in the RPE65 gene. The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
Maybe. Make sure you are in the FFB patient registry (https://www.myretinatracker.org/). Spark called us out of the blue to schedule a test to determine if it will help us. I assume they are reviewing the registry and contacting candidates for treatment that they find there?
He would need genetic testing to determine which gene is causing his RP. If that is the RPE65 gene then he may be a candidate
What wonderful and exciting news. Best Christmas gift ever.
Fascinating!
Fantastic news!!! Christmas miracle
How do my dad and I get this done to help our RP
Hi Skylar,
LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
My 2 sons suffer from RP. How’d we get treatment?
Hi John,
LUXTURNA is for people with mutations in RPE65 gene. But the following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
This is amazing!! Is this only for rpe65 Gene?
Hi Carri,
Yes, LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
God bless Sparks and the Foundation Fighting Blindness and all those dedicated people who worked on the research for and development of Luxterna. My wife has lost all her vision due to RP. This brings us hope.
HOW DO I FIND A PLACE IN NORTHERN CALIF. WHERE I CAN GET THIS LUXTURNA
GENE THERAPY???
Hi Jo Anne, to learn more about access to LUXTURNA visit: https://luxturna.com/. Keep in mind that LUXTURNA is only for people with mutations in the gene RPE65.
With two brothers with RP it is a hopeful sign their vision will be returned!
congratulations!! I am possible to interest participation., but cost really is ouch!!! I am recipient of government check , I am deaf and with retina pigmontoea. I want to be free as normal people what I was used in my youth. I am current 55 years old.
Hi Johnny,
LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease. The cost has not yet been announced. Any specific questions about pricing, patient access programs or the availability of the treatment should be referred to Spark Therapeutics. Spark’s website address is: http://www.SparkTX.com – patient inquiries number is 1 833 SPARK PS (833 772 7577).
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
1. What is the cost?
2. What age groups are targeted?
3. At what stage of RP would treatment be considered?
4. If already on organ transplant antirejection meds, would treat be advisable?
LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease. The cost has not yet been announced. Any specific questions about pricing, patient access programs or the availability of the treatment should be referred to Spark Therapeutics. Spark’s website address is: http://www.SparkTX.com – patient inquiries number is 1 833 SPARK PS (833 772 7577).
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
My brother has RP. He is 53. What is the costs. And what criteria qualifies a candidate for treatment?
Hi Rich,
LUXTURNA is only for people with mutations in the RPE65 gene. He needs genetic testing to identify the mutated gene causing his RP. The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest he register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
what wonderful news.I hope this works for macular degeneration some day soon.
Good news! Is there any news for Macular
Degeneración ?
FFB is funding several projects for AMD. You might also visit http://www.clinicaltrials.gov to learn about emerging treatments for AMD.
My daughter is recntly diagnosed with RP.She is 14 year. Very intellegnt.She is my hope. Please let me know is there is hope for her.
To learn about LUXTURNA, the first FDA-approved gene therapy for the retina visit: https://luxturna.com/. Keep in mind that LUXTURNA is only for people with mutations in the gene RPE65.
Any plans for gene therapy for those with LHON? My husband connected Lebers at the age of 62 and is desperate for help. His mutation is 11772, that most common mutation.
Hi Kandy,
There are several clinical trials for LHON. Visit http://www.clinicaltrials.gov (search on LHON) for more information on those studies.
It’s wonderful.maybe my son will see again!thank you for all your work
Wonderful and best mankind on the earth.
Proud and how amaising are the científics ti find the ways to resolve a problem.
Very thanks for your dedication
OMG!!!!! My mom had RP and so do I!!!! I’m sooooooooooooo happy! Thank you researchers!! Your amazing, thank you for your hard work!!! Where can I sign up to have it done???? 2018 hopefully looking better. So thankful!
Hi Lynne,
LUXTURNA is for people with mutations in RPE65 gene. It is important for people to get genetically tested so they learn what gene mutation is causing their disease.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
Will this treatment be helpful for people with dry macular degeneration?
Hi Elizabeth,
No – it isn’t for dry AMD, but this will help open the door for development of additional vision treatments for other diseases. Visit http://www.clinicaltrials.gov and search on age-related macular degeneration to learn about clinical trials for emerging dry AMD therapies.
I am excited about the identity of RPE65 and the Luxturna treatment. I am 66 yrs. ld and have RP autosomal dominant. This will help me and my family members who inherited this disease.
Wonderful, hope this treatment will be available in more hospitals soon.
My wife and her sister both suffer with R.P. and desperatly want treatment
Very thrilled to hear about this miracle cure. Thanks to the foundation who has been working on this research for decades . Many people will be blesse . Great Christmas gift. God bless you all.
Like to know more about it.
I have lost all of my sight to R P, hope your hard work will some day restore my sight, God bless each of you.
My son was diagnosed with X – linked retinoschisis a few years ago. Such wonderful news about this new trial LUXTURNA! Prayers that it will help many people!
With committed Researchers and Organizations we have been given hope that maybe someday our vision loss will be restored. To know that our children who may have inherited this eye disease will be able to live as it is meant to be without an eye disease holding them from having a full and purposeful life is a BLESSINGS to dos many! Thank you!! Whether I’m put on a waiting list it will be worth the wait!!
BLESSINGS TO ALL!
MERRY CHRISTMAS AND HAPPY BEW YEAR TO ALL!!!
This could change mind and my entire families life. I hope this becomes a reality.
Great news indeed. Is this therapy applicable for ABCA4 gene as well? Please let me know. Both my daughters have stargardts.
This treatment is for the RPE65 gene. There is an ABCA4 gene therapy trial in Portland, Oregon. Visit the following page to learn more about the trial and coordinator contact information: https://clinicaltrials.gov/show/NCT01367444
You can search this site for other clinical trials, as well – search on ABCA4.
The best Christmas gift ever.
The Best Christmas Present my son could have ever received!! A million thank you’s!!!
This is amazing news,
I have two children with stargazers both now in their early 20s and both have suffered with day to day struggles for many of those years.
I can now give them hope !!
From a mother who has been strong and encouraging every day to not give up and be the best they can be considering their vision impairment
wonderful – i hope this will help many: will this genetic treatment help stroke related retinitis – my right eye went blind from cva some years ago, but has come back to 40% over the years. ???
Hi John,
LUXTURNA is for people with retinal disease caused by mutations in the gene RPE65, not for your retinal condition. We suggest contacting your retinal specialist to learn more about potential therapies for your condition.
Dear Sir,
Emmencely thankful to one and all responsible for this breakthru scientific service to humanity since centuries.
Congratulations and A MERRY CHRIST MASS to you all. Now millions of eyes can see Jesus in front of them.
God is so good! His guidance has led science to make this fantastic discovery. My 79 year old husband has RP….he had to retire at 47 years old. Him being able to see again would be wonderful, but if not, this should help millions of people.
I pray this gets to many retinal doctor’s across the U.S.A., as it is costly for some of us to travel. I also pray Medicaid, and Medicare will pay for treatment. I have RP, and only see out of one eye now. Please help, and God bless all involved.
Both of my sons have RP. They are now 32 and 35. What would the next step be to help them?
Hi Mary,
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
This is amazing news to everyone affected by RP. I hope this treatment will be available worldwide ASAP.
I have retinitis Pigmentosa and have dealt with it most of my adult life. My vision is not totally gone but I can feel it everyday diminishing. To have a medical advancement like this is amazing and I would love to be a part of it. I had a brother and older sister with the same disease. My sister had to rely on a seeing eye dog and it was really sad to watch her. It would be a dream to be able to have medical help and not go totally blind.
The following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
Spark contacted us to schedule a test to determine whether we can be helped by this treatment. I assume that they found us through the FFB patient registry. If you haven’t enrolled in the database yet, I suggest you go here: https://www.myretinatracker.org/ and register.
When and where is this available. I gave 2 son’s with RP.
Hi Linda,
LUXTURNA is for people with mutations in the gene RPE65. Your sons need to be genetically tested to determine which gene is causing their RP. If you (they each) register at http://www.myretinatracker.org, you can receive notifications of FFB’s genetic testing program.
My son , 26 years old, has a RP, gêne USH2A. Is it possible to
Know if this treatment will be for him or anything is coming soon.
Best gift for ever…
LUXTURNA is for people with mutations in the gene RPE65. But the following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
FFB is also conducting a natural history study for people with USH2A mutations:
http://www.blindness.org/blog/index.php/ffb-cri-launching-natural-history-study-for-people-with-ush2a-mutations/
I have RP. How does one become a participant in this?
LUXTURNA is for people with mutations in the gene RPE65. You need to be genetically tested to determine which gene is causing your RP. If you register at http://www.myretinatracker.org, you can receive notifications about FFB’s genetic testing program.
i have 2 sons who have RP. How do we get treatment?
Hi John, LUXTURNA is for people with mutations in the gene RPE65. But the following emerging therapies are cross-cutting and may provide benefits to people with many forms of RP:
http://www.blindness.org/blog/index.php/jcyte-reports-results-for-phase-12a-clinical-trial-for-retinal-cell-treatment/
http://www.blindness.org/blog/index.php/sparingvision-formed-to-advance-sight-saving-protein-for-rp/
http://www.blindness.org/blog/index.php/foundation-investing-in-drug-to-slow-many-forms-of-rp/
We also suggest you register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and clinical trials for which you may be eligible.
Hello Mr Ben Shaberman
I have lost all of my sight to RP, hope your hard work will some day restore my sight, God bless each of you Mr Ben Shaberman and Foundation Fighting Blindness .
This could change mind and my entire families life. I hope this becomes a reality.
My husband, would low vision be interested in this treatment since he (and one Brother and 2 daughter) has Retinitis Pigmentosa. Please keep us informed as to when this treatment would be available. We are in the iran-tehran area.
Please help,
Mr.Moji.E
I have two sons in there thritys with Retinitis pegmentosa usher syndrome,it would be a miracle if they can get there sight back.i pray that will happen soon.
I just read in the Colorado Springs Gazette that Spark will be charging $425,000 per injection for a total of $850,000 for bilateral injections. These costs are exhorbitant and will not be covered by insurance. And what happened to the $10 million that FFB contributed to start the effort and my donations over the years to contribute to FFB and allow Spark to gouge the public?
Hi Daniel,
Spark is working with insurance companies to gain coverage for the treatment. Spark is doing all it can to provide access to the treatment for those who need it. Visit https://luxturna.com/take-the-first-step to learn more about gaining access to LUXTURNA. FFB’s $10 million investment went to early research that made the clinical trials for LUXTURNA possible.
Will this save the eyesight ..of people w/cone rod dystrophy? My daughter was diagnosed / this when she was 10 yrs old..
Hi Lenny,
LUXTURNA is not for people with cone-rod dystrophy. She needs genetic testing to determine what mutated gene is causing her vision loss. That will help her determine which emerging therapies may be relevant. We suggest she register at http://www.myretinatracker.org to qualify for free genetic testing (coming soon) and learn about clinical trials for which she may be eligible.
Lost the vision of my left due to a bacterial infection. I also have glaucoma in both eyes. Some vision in left eye but not much no side vision. Would a treatment like this help me?
LUXTURNA is for a very specific inherited retinal disease. We suggest you contact a glaucoma specialist.
Would love to know if I could be a candidate. This is an answered prayer for so many.
Hi Susan, LUXTURNA is specifically for people who have mutations in the gene RPE65. You need to be screened with a genetic test to determine which gene is associated with your vision loss. Sign up at http://www.MyRetinaTracker.org to make yourself eligible for free genetic testing.
Wonderful news! I have RP-ushers. But I don’t know what type. I’ve been looking to see king for genetic testing but not having any luck. Thank you for your good work. Hoe can we get more infor I want my sight back
Hi Linda,
If you register at http://www.MyRetinaTracker.org, you can make yourself eligible for free genetic testing. That is a good place to start.
Where do I get this treatment. I have last stages of Stargardt disease, my center vision is 20/400 in both eyes. Can anyone point me in the right direction as where to begin? Please let me know if you do.
Hi Gary,
This treatment is not for Stargardt disease. Visit http://www.clinicaltrials.gov to learn more about clinical trials for Stargardt disease. (You can search on “Stargardt disease.”)
I am suffering from RP and want to get this treatment done for my self. what is the procedure. also since I am from india, is the treatment can be performed here? do let me know, so that I can make the arrangement to come over USA and get the treatment.
Thanks
First, you should understand that this therapy is only for people with mutations in the gene RPE65. There are many genes that can cause RP – you need to have a genetic test to determine which mutated gene is causing your disease.
We are aware of the following clinics in India, which may be helpful:
Shankara Netralaya, Chennai
L. V. Prasad Eye Institute, Hyderabad – They have branches in different states
Aravind Eye Hospital, Madhurai
All India Institute of Medical Sciences- New Delhi
If you are interested in a clinic in a different location, contact one of these clinics and ask them for a suggested clinic in your region.
Steps in the right direction! Hats off to all in America for establishing FFB and all the fantastic people raising funds for research. I live in the U.K. and have an inherited eye disease as have many other people in my family, mother, sister, aunts, cousins etc and like many people with RDD it has taken years to get a diagnosis, from when I was a teenager in the 1980 s I was told I have RP then to be told I have AMD and recently I have been informed that I have a rod/cone like dystrophy. I have also been gene tested and the result is that I have a mutation in the GUCY2D gene. If any of your researchers are looking in this area and need a case study or anything that may help I would be more than happy to pass my medical history/records over for perusal. Thanks to all involved.
OMG!! My daughter was diagnosed with Stargardt disease last Spring. I have been following the research and blogs since then. Now there is real, tangible hope. I pray we can provide this for her. Thank God Almighty…
I was diagnosed with RP 9 years ago. It seems that my vision is worsening at a quicker pace these last several months. Hopefully this is the answer to our prayers.
what a great news genetic engineering will be developed
This is great news but I am worried about the cost! I am a mother with Stargardt’s Disease and Congenital Stationary Night Blindness. My life would be so different. I be able to support my two kids much better. I work full-time and have a child who is Down Syndrome. I am thankful for love ones, but I am very independent. Crossing streets with a little one would be less stressful. Please tell me this would be affordable…
Hi Tonnya, Spark Therapeutics has worked very closely with payers to allow commercially insured patients who seek treatment within network to be treated at no or near no patient out-of-pocket expense. If you have any further questions, please feel free to visit their Patient Support Services website (www.mysparkgeneration.com) or call their patient inquiry number at 1 833 SPARK PS (833 772 7577). Hope this helps!