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The company is evaluating whether the drug, which targets cardiac complications, might be used treat patients with other ...

Evan McMenamin said his brother Travis, "never lets it get him down" despite the "incredible challenges" he faces ...

Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...

At just 15 years old, Ragheeb has already made a tremendous impact on his community as an ambassador for Capes for Kids, an ...