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

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 ...
Seeking Alpha

Orchard Therapeutics Outlines U.S. Launch Plans for Lenmeldy™ (atidarsagene autotemcel), the Only Approved Therapy for Children with Early-onset Metachromatic Leukodystrophy

--Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the details of its U.S.
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Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the ...

TOKYO and LONDON and BOSTON, March 18, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients ...
KTVZ

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

(CNN) — 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 ...
KWWL

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

Babies with MLD develop normally for a time and but then typically begin to lose the ability to walk and talk around age 2. The disease advances rapidly, causing children to deteriorate into a ...
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 ...
WSIL-TV

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

About 40 children are born with MLD in the US each year. The wholesale cost isn’t usually what patients pay, but it’s a cost that’s considered and shouldered by public and private health insurance ...