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Japan’s MHLW grants orphan status to Kyowa and Orchard’s MLD therapy

Japan's Ministry of Health, Labor and Welfare (MHLW) has granted orphan regenerative medicine product designation to Kyowa ...
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 ...
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 ...

Kyowa Kirin and Orchard Therapeutics Announce OTL-200 Granted Orphan Regenerative Medicine Product Designation for Early-onset MLD in Japan

First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the Kingdom of Saudi Arabia –TOKYO, LONDON and ...
Morningstar

Orchard Therapeutics Appoints Robin Kenselaar to Chief Commercial Officer and Announces Multiple Business Updates Pertaining to its Approved HSC Gene Therapy for MLD | Morningstar

TOKYO and LONDON and BOSTON, Feb. 12, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced the appointment of Robin Kenselaar as chief commercial officer. In this role ...
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 ...
Business Insider

Orchard Therapeutics Appoints Robin Kenselaar to Chief Commercial Officer and Announces Multiple Business Updates Pertaining to its Approved HSC Gene Therapy for MLD | Markets ...

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 ...