This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

BEDFORD, Mass., Oct. 21, 2019 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of preclinical data that support its ...

(RTTNews) - The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel) gene therapy to treat children with early-onset metachromatic leukodystrophy or MLD. Lenmeldy, formerly ...

Metachromatic leukodystrophy (MLD) is an inherited, autosomal recessive lysosomal storage disease (LSD) caused by a deficiency in the lysosomal enzyme arylsulfatase A (ASA), which catalyzes the ...

LENMELDY™ is the first and only disease-modifying intravenous infusion proven to extend life expectancy in pre-symptomatic late infantile (PSLI) patients and mitigate the cognitive and/or physical ...

Shares have lost a third of their value over the past year. HSC-based gene therapy Libmeldy is approved in the EU for MLD with a ~$3.5M price tag. Incidence could be higher than initial estimates.

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 ...

Share on Pinterest Scientists have managed to create a treatment for the rare genetic disorder MLD. smolaw11/Getty Images Doctors in the United Kingdom have used a revolutionary gene therapy to cure a ...

First U.S. patients being treated with Lenmeldy™ (atidarsagene autotemcel) in a commercial setting; other launch efforts progressing well following Food and Drug Administration approval last year ...

TOKYO, LONDON and BOSTON, May 13, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced a publication on long-term safety and efficacy outcomes for Lenmeldy™ ...