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Displaying 61–70 of 229 diseases results
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Research News
The company has also launched a natural history study of patients to inform the planned clinical trial.
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Staying True to Music, Family, and Advocacy
Beacon StoriesMusic duo Andrew & Polly have earned a GRAMMY nomination for their children’s album “Ahhhhh!” while also championing disability awareness and inclusivity. Andrew’s experience with Stargardt disease fuels their advocacy, inspiring others through their music, podcast, and dedication to research and community support.
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Sepul Bio Launches Phase 2b Clinical Trial for USH2A RNA Therapy
Research NewsKnown as ultevursen, the USH2A RNA therapy was licensed from ProQR.
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Eye on the Cure Podcast | Episode 77: Dr. Bob Bell
Eye on the CureDr. Bell, the CSO of Ascidian Therapeutics, talks about STELLAR, the company’s Phase ½ clinical trial for its emerging RNA-rewriting therapy for Stargardt disease (ABCA4 mutations).
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Eye on the Cure Podcast | Episode 76: The Lemay-Pelletier Family
Eye on the CureWhen the Lemay-Pelletier family from Montreal learned that three of their four children were losing vision from retinitis pigmentosa, they made the bold decision to spend 15 months traveling the world to give their kids as many visual memories as possible.
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Research News
Strengthening research in retinal disease with record-breaking $19.8 million investment in innovative projects targeting vision loss worldwide.
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Atsena’s LCA1 Gene Therapy Licensed by Nippon Shinyaku for Markets in US and Japan
Research NewsThe companies are planning a Phase 3 clinical trial for the promising gene therapy.
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BlueRock’s Photoreceptor Cell Therapy to Move into Clinical Trial
Research NewsThe Foundation funded Dr. David Gamm for development of the induced pluripotent stem cell approach to be used in the trial.
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Kiora Receives Authorization for Phase 2 Clinical Trial of its Photoswitch for Advanced RP
Research NewsThe 36-participant trial will take place at multiple sites in Australia.
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Eye on the Cure Podcast | Episode 75: Sharon King
Eye on the CureSharon King, co-founder and president of Taylor’s Tale, talks to host Ben Shaberman about her family’s journey when her daughter Taylor was diagnosed with Batten disease.
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