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Posts tagged usher syndrome

FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A

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The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB-CRI will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a, toward a Phase 1/2 clinical trial during 2018. ProQR plans to issue the initial data report for the clinical study in 2019.
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Clinical Trial to Launch for System Combining Optogenetics and Eyewear

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gensightThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.

The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
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ProQR Doses First Participant in Its LCA10 Therapy Clinical Trial

ProQR, a biotech company in the Netherlands, has treated its first patient in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.

The Phase 1/2 trial will enroll six children and six adults who have the mutation. Participants will receive a dose in one eye every three months for a year. The treatment is delivered through an injection into the vitreous, the gel-like substance in the middle of the eye.
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FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
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Artist with Usher Syndrome Excited to Register on My Retina Tracker to Drive Retinal Research

Artis Dana Simon at work.In a post at, artist Dana Simon describes her experience with My Retina Tracker, a free and secure online registry for people with inherited retinal diseases. My Retina Tracker provides researchers with invaluable information that helps them study retinal diseases, and informs patients when their profile matches clinical-trial criteria. Examples of Dana’s artwork can be found on her website.

Back in Baltimore – VISIONS 2016, FFB’s National Conference, Returns to Its Founding City

Baltimore's Inner HarborFor the second year in a row, the Foundation Fighting Blindness’ national conference — this year titled VISIONS 2016 — will take place in FFB’s founding city of Baltimore, Maryland, June 30-July 3. More than 500 people are expected to attend the event, which will offer dozens of research- and lifestyle-focused sessions designed for people who are visually impaired and sighted as well.
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Traveling, Whether It Is Dark or Light

Moira Shea walking with her guide dog, Finnegan, in Barcelona, Spain.In November of 1971, the same year the Foundation Fighting Blindness (FFB) was established, I was diagnosed with Usher syndrome, a disease that causes both hearing and vision loss. I was 15 at the time, and my parents and I quickly became involved with FFB, and over the past 45 years, I’ve been able to meet many others dealing with the fear of blindness.
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For Rare Disease Day – The Many Benefits of Genetic Research

Rare Disease Day logoIt’s apropos that Rare Disease Day 2016 will be held on the rarest day on the calendar—Leap Day, February 29.

However, collectively, rare diseases are not uncommon. About 30 million Americans, nearly 10 percent of our population, are affected by one of 7,000 rare diseases. They’re an important public health issue, making it incumbent upon us to work hard to eradicate them.
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2015 Top 10 Retinal-Research Advances

Researcher in a labThe Foundation Fighting Blindness’ scientists, donors and volunteers made 2015 an outstanding year in our fight against blindness. As I tabulated the year’s top 10 research advances—all made possible through FFB funding—I realized that eight are for clinical trials of emerging therapies that are launching or underway.
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VISIONS 2015, Faces of VISIONS – Carol Brill

Carol Brill“If one person’s giving me a hard time, I’m not going to let him ruin my day,” says Carol Brill. “I always remember there are seven billion other people in the world who can make me happy.”
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