Researchers link early cone dysfunction in RPGR-related retinal dystrophy to high myopia, with timing of degeneration ...

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 ...

More information: Michel Michaelides et al, Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study, The Lancet (2025). DOI: 10. ...

The loss of UBAP1L gene function appears to be associated with nonsyndromic retinal dystrophy, according to research published in JAMA Ophthalmology.“Inherited retinal dystrophies (IRDs) present ...

Retinal structure and brain activity in response to light was also measured. And sure enough, all 11 children treated so far have seen “meaningful responses” to the therapy.

Odylia Therapeutics, a nonprofit (501(c)3) biotechnology company dedicated to advancing treatments for rare diseases, today announced its latest pipeline project: a unique and innovative co ...

BARCELONA, Spain — In this Healio Video Perspective from the Euretina congress, Bart Leroy, MD, PhD, speaks about the overlapping features of uveitis and inherited retinal dystrophies.The ...

As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subre ...

Viralgen and Axovia Therapeutics are joining forces to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with ...

Gene therapy using a recombinant adeno-associated viral vector carrying the AIPL1 gene improved visual acuity and preserved retinal structure in young children with AIPL1-associated retinal dystrophy.