Inherited retinal dystrophy (IRD) isn’t one disease. It’s many different ones that cause your retina to break down and stop working. They can make you color-blind or night blind. Others can lead to ...

FDA authorization under an IND permits first-in-human phase I/II testing of SVT-001 in familial drusen, focusing on safety ...

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 ...

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 ...

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 ...

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, ...

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 ...

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 ...

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 ...

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 ...