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Displaying 1–10 of 196 diseases results
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ARVO 2025 Highlight: J&J’s XLRP Gene Therapy Didn’t Meet Primary Endpoint in Phase 3 Clinical Trial
Research NewsDespite missing the primary endpoint, the treatment improved vision for some patients.
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Research News
The company is planning a Phase 2/3 clinical trial for the therapy in late 2025.
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Research News
Part B of the trial will enroll nine adult patients as well as three pediatric patients.
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Research News
The company is currently enrolling patients in its Phase 2/3 VISTA Clinical Trial.
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Eye on the Cure Podcast | Episode 85: Cindy Elden
Eye on the CureCindy Elden talks about her journey with Usher syndrome type 3 (USH3), the decades-long work and persistence of her non-profit, and the clinical trial it launched for an USH3 small molecule therapy.
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Beacon Stories Wellness
Rob Howden’s journey with AMD and GA truly shows that individuals can take control of their vision loss journey and find strength within their community. His story emphasizes the importance of proactive treatment, taking care of your mental health, and the invaluable support of friends and neighbors.
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Living With Usher Syndrome: Practicing, Prioritizing, and Protecting Your Mental Health
Living and Thriving WellnessIn this deeply personal reflection, Rebecca Alexander, LCSW-R, MPH, RYT, shares the emotional journey of living with Usher syndrome—not as a single moment of diagnosis, but as an ongoing evolution of identity, loss, and resilience. Drawing from her experience as a psychotherapist, she offers powerful insights and coping tools for navigating grief, preserving connection, and embracing the full complexity of the human experience.
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Atsena Receives $150 Million in New Financing to Advance XLRS and Preclinical Gene Therapy Programs
Research NewsThe company has reported encouraging results from its LCA1 and XLRS gene therapy clinical trials.
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Beacon Stories
Danielle has been navigating her retinitis pigmentosa for half of her life, but she’s never let it hold her back. Her journey with RP underscores the importance of perspective, community of her friends and family’s support, and the relentless pursuit of treatments and cures.
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Eye on the Cure Podcast | Episode 83: Michel Michaelides
Eye on the CureProfessor Michaelides talks about the extraordinary vision improvements for young blind children receiving gene therapy for LCA4
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