The FDA has approved the first-ever gene therapy for Wiskott-Aldrich syndrome, marking a major milestone for the rare disease community. While this treatment is not for vision loss, it represents important progress for gene therapy as a whole — and brings renewed hope for families awaiting treatments for inherited retinal diseases like BBS and retinitis pigmentosa.

Patient advocacy is now one of the most powerful drivers of progress in retinal gene therapy. Learn how nonprofit involvement, family voices, awareness, and fundraising are accelerating research for Bardet-Biedl Syndrome and inherited retinal diseases — and why this work matters now more than ever.

The latest innovations in gene therapy — from optogenetics to engineered AAV capsids to AI-driven breakthroughs — are rapidly accelerating progress toward treatments for Bardet-Biedl Syndrome and other inherited retinal diseases. Here’s what these advances mean for families urgently awaiting hope.

We recently attended the Gene Therapy for Ophthalmic Disorders Summit, where leading researchers and clinicians shared promising advances in retinal gene therapy. From new vision endpoints to breakthroughs in delivery methods and patient advocacy, the conference highlighted real progress toward treatments for BBS, RP, and other inherited retinal diseases. Here’s what we learned — and why it matters for families urgently waiting for hope.

See this week’s uplifting reviews and experiences from donors whose kindness and belief in our mission fuel everything we do.