The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early ...

Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of Philadelphia, ...

Finding showed treatment with Lenmeldy significantly extended severe motor impairment-free survival in children with PSLI MLD compared with untreated natural history children. The Food and Drug ...

Please provide your email address to receive an email when new articles are posted on . Metachromatic leukodystrophy is an incurable rare genetic disease. At 5 years of age, 71% of children given ...

Get the Well Enough newsletter with Harry Bullmore for tips on living a healthier, happier and longer life Get the Well Enough email with Harry Bullmore A scientist who diagnosed her own daughter with ...

The Food and Drug Administration on Monday approved a gene therapy for metachromatic leukodystrophy, a devastating genetic disorder that eats away at affected children’s neurons. The medicine, ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

BEDFORD, Mass., Aug. 11, 2022 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the details of HMI-204, its optimized, in vivo, one-time gene ...

Currently, there are no approved treatments for MLD, a lysosomal storage disease caused by a deficiency in arylsulfatase A. The following article features coverage from the 17th Annual WORLDSymposium ...