Please provide your email address to receive an email when new articles are posted on . The loss of UBAP1L gene function appears to be associated with nonsyndromic retinal dystrophy, according to ...

MeiraGTx Holdings plc announced promising results from a first-in-human study published in The Lancet, involving a gene therapy treatment for children with AIPL1-associated retinal dystrophy, ...

LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of ...

Odylia Therapeutics Inc. has announced it is working under a codevelopment partnership with the NPHP1 Family Foundation to create an AAV-based gene replacement therapy for retinal dystrophy caused by ...

Scientists at the National Institutes of Health (NIH) and their colleagues have identified a gene responsible for some inherited retinal diseases (IRDs), which are a group of disorders that damage the ...

ATLANTA, May 5, 2025 /PRNewswire/ -- Odylia Therapeutics, a nonprofit (501(c)3) biotechnology company dedicated to advancing treatments for rare diseases, today announced its latest pipeline project: ...

Collaborative effort will bring hope to BBS patients suffering from retinal degeneration through this investigational gene therapy. Bardet-Biedl Syndrome (BBS) is a genetic condition that causes multi ...

Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with rAAV8.hRKp.AIPL1 All 11 children treated with rAAV8.hRKp.AIPL1 between 1 and 4 years old were legally blind ...