Too often, regulatory tools authorized to speed patient access to breakthrough treatments are being used in ways that even prevent approval,” writes Kat Bryant Knudson.

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North ...

MILAN, Italy, July 31, 2026 – Italfarmaco S.p.A. today announced a managed entry agreement with the Georgian Ministry of Health for givinostat ...

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

KYOTO--Utilizing genome editing technology, a team of researchers has developed a much more effective treatment for a certain ...

An FDA advisory committee voted 9-3 against the efficacy of deramiocel for treating cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive ...

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the company will present data on sevasemten, an ...

At a contentious meeting with possible implications for other rare disease drugmakers, outside experts ultimately determined that Capricor didn't prove deramiocel's benefits in Duchenne-related ...

Researchers combined advanced imaging techniques and theoretical physics to observe and explain how nanoclusters of the protein emerin form inside living cells. The study uncovers the molecular 'rules ...

Dublin, July 16, 2026 (GLOBE NEWSWIRE) -- The "Duchenne Muscular Dystrophy Treatment Market Outlook 2026-2034: Market Share, and Growth Analysis by Therapeutic Approach (Molecular-Based, Mutation ...