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 ...
Monthly Prescribing Reference

Lenmeldy Approved for Children With Metachromatic Leukodystrophy

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 ...
Nasdaq

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the ...

One-time treatment with Lenmeldy has shown the potential to restore enzymatic function to stop or slow disease progression, with up to 12 years of follow-up (median 6.76 years) TOKYO and LONDON and ...
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

Gene therapy delivered early can help children with rare neurodegenerative disease retain motor and cognitive functions

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 ...
Reuters

US approves first gene therapy for children with rare genetic disease

March 18 (Reuters) - The U.S. health regulator on Monday approved UK-based Orchard Therapeutics' gene therapy to treat children with metachromatic leukodystrophy (MLD), making it the first approved ...
AOL

A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access

A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s most ...
News Medical

Gene therapy offers hope for children born with metachromatic leukodystrophy

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 ...
The Scientist

The Philadelphia Family Helping to Drive Research on MLD

Children with MLD cannot produce arylsulfatase-A, an enzyme that breaks down sulfatides. So the compounds accumulate in the nervous system, where they attack the myelin sheaths that cover nerve axons, ...
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.