None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Sarepta Therapeutics (NASDAQ:SRPT) stock draws an Overweight rating at Wells Fargo, based on commercial prospects for ...

One Little Boy. One Big River. One Chance to Save His Life. “It’s just one little boy,” they say. But to everyone who knows 6-year-old William Jackson, he is everything. Diagnosed with Duchenne ...

Zydus Lifesciences has secured final approval from the US Food and Drug Administration (USFDA) to manufacture Jaythari (Deflazacort) tablets in four strengths - 6 mg, 18 mg, 30 mg, and 36 mg.

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

Zydus Lifesciences has received final approval from the United States Food and Drug Administration (USFDA) to manufacture Jaythari (Deflazacort) Tablets, 6 mg, 18 mg, 30 mg, and 36 mg (USRLD: Emflaza ...

Keros Therapeutics halted phase 2 of TROPOS, but KER-065 shows promise in DMD and financial stability ensures growth potential through 2029. Read why I rate KROS as a Hold.