Forbes

From Research To Reality: Gene Therapy For Metachromatic Leukodystrophy

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 ...
Nasdaq

Homology Medicines Presents Data from Investigational PKU and MLD Gene Therapy Programs that Demonstrate Preclinical Proof-of-Concept for Potential One-Time Therapies

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 ...
Medscape

FDA OKs First Gene Therapy for Metachromatic Leukodystrophy

The US Food and Drug Administration (FDA) has approved the first gene therapy for metachromatic leukodystrophy (MLD), a rare and debilitating genetic disease affecting the brain and nervous system.
Medical News Today

How a gene therapy cures infants with fatal genetic disease MLD

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 ...
Nasdaq

FDA Approves First Gene Therapy For Kids With Early-onset MLD

(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 ...
Medical Xpress

First gene therapy for children with metachromatic leukodystrophy approved by FDA

The U.S. Food and Drug Administration has approved Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy ...