The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

Like Duchenne muscular dystrophy, Becker muscular dystrophy typically affects only males (1 in 30,000) and causes heart problems. Disease severity varies. Those with Becker can usually walk into ...

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants with Duchenne muscular dystrophy (DMD) at 90 days post treatment ...

Happy Friday. Today, we talk about the stem cell fire sale at CIRM, and about the FDA’s move to release a limited batch of ...

Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

Duchenne Muscular Dystrophy: Predictors of Decreased Pulmonary Function Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients ...

Duchenne muscular dystrophy is the most common and severe form of the disease. It usually starts when a child is between ages 2 and 5. Symptoms of Duchenne muscular dystrophy include: ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus. Neurology , 2025; 104 (11) DOI: 10.1212/WNL.0000000000213604 Cite This Page : ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, ... Duchenne muscular dystrophy: Five trials to watch. Joshua Silverwood . Wed, Mar 26, 2025, 2:59 PM 7 min read.