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 ...
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 ...
Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy ...
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, ...
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 ...