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, ...

(RTTNews) - MeiraGTx Holdings Plc. (MGTX), a clinical-stage genetic medicines company, on Friday announced the progress in its gene therapy program for Leber Congenital Amaurosis 4 or LCA4, a severe ...

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 ...

Bestrophinopathy and Vitelliform Macular Dystrophy represent a spectrum of inherited retinal disorders predominantly associated with mutations in the BEST1 gene. These conditions are characterised by ...

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 ...

Please provide your email address to receive an email when new articles are posted on . BARCELONA, Spain — In this Healio Video Perspective from the Euretina congress, Bart Leroy, MD, PhD, speaks ...

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: ...

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 ...