Business Insider

Sarepta Therapeutics Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Functional Improvements 18-months After ...

-- Continued functional improvements wereobserved at 18 months in the low-dose cohort-- -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration -- -- ...
Seeking Alpha

BridgeBio to Host Limb-girdle Muscular Dystrophy Type 2I/R9 Investor Webinar on Friday, July 11th at 8:00 am ET

PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced ...
Business Wire

IND for ATA-100, a Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today ...
Morningstar

Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9)

Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9) Designation supports development of CRD-003, an ...
WKRG

AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

RESEARCH TRIANGLE PARK, N.C. , Aug. 3, 2023 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, ...
Business Wire

Sarepta Therapeutics Completes Enrollment in EMERGENE, a Phase 3 Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that enrollment and dosing is complete in ...
Business Insider

Muscular Dystrophy Association Announces More Than $2.7 Million For Research Grants to Advance Breakthroughs Across Neuromuscular Diseases

New York, Dec. 10, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) today announced more than $2.7 million in new research grants to advance groundbreaking discoveries across multiple ...
Nasdaq

SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program

Shares of Sarepta Therapeutics SRPT nosedived 35.9% on Friday following the death of a patient dosed with one of its experimental gene therapies. The company confirmed that a 51-year-old, non-ambulant ...