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 ...

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 ...

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 ...

Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a ...

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 ...

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 ...

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 ...