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 ...

Sentinel participant showed OPGx-BEST1 was well tolerated with no ocular inflammation, treatment-related adverse events, or dose-limiting ...

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, ...

This story is part of a series on the current progression in Regenerative Medicine. This piece is part of a series dedicated to the eye and improvements in restoring vision. In 1999, I defined ...

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 ...

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw improvements ...

CHICAGO — Children with a rare, inherited retinal disorder who were treated with the first Food and Drug Administration-approved gene therapy had noticeable improvements in vision that may be related ...

Alkeus Pharmaceuticals, Inc., a biopharmaceutical company dedicated to preserving the sight of individuals impacted by retinal diseases, today announced that results from its clinical study of ...

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 ...

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 ...