The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial ...

Duchenne muscular dystrophy (DMD) is a severe genetic condition characterized by progressive muscle weakening. People with DMD typically only live into their 20s, but life expectancy has been ...

New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular ...

Duchenne Muscular Dystrophy affects 12,000 to 15,000 children and young adults in the United States and about 300,000 worldwide. It's caused by a mutation in the dystrophin gene, which makes a ...

Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to approach physical activity and exercise. While physical activity may help ...

Aug. 27, 2020 — Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular ...

Duchenne muscular dystrophy Dr. Edwin P. Ewing, Jr./CDC P at Furlong was sitting in her home office in Middletown, Ohio, last Thursday, refreshing a Food and Drug Administration web page ad ...

Regenerating muscle in Duchenne muscular dystrophy: Age matters. ScienceDaily. Retrieved June 2, 2025 from www.sciencedaily.com / releases / 2014 / 04 / 140414091747.htm.

Eight therapies are approved for Duchenne muscular dystrophy (DMD) in the U.S., and all have been approved since 2016. "It took 30 years from the discovery of the gene associated with Duchenne ...