The newly approved therapy Vyondys 53 (golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD)…
Articles by Marta Figueiredo, PhD
The U.S. Food and Drug Administration has conditionally approved Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD)…
An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (DMD) in…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s…
Parent Project Muscular Dystrophy (PPMD) announced the screening of a first baby for Duchenne muscular dystrophy (DMD) within the organization’s…
Researchers have identified a new set of blood biomarkers of Duchenne muscular dystrophy (DMD) and of response to standard…
Sarepta Therapeutics is partnering with Roche to accelerate the development and expand the availability of SRP-9001 —…
Capricor Therapeutics’ vesicle-based therapy CAP-2003, derived from the company’s proprietary cardiosphere-derived cells (CDCs), may be an effective therapy…
Microdystrophin gene therapy robustly induces the production of a shorter, but functional, version of the dystrophin protein and reduces muscle…
A new approach to CRISPR-Cas9 gene-editing technology, called myoediting, successfully restored dystrophin production and contraction force in heart muscle cells…