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

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...

Listen to this article The Virginia Home plans to build a $128 million, 75-acre campus in Hanover County, according to a Thursday announcement by the nonprofit, which serves adults with irreversible ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

Tony, from Skellingthorpe, Lincoln, was diagnosed with myofibrillar myositis and anti-SRP immune-mediated necrotizing ...

WVE-007 is a novel, long-acting GalNAc-siRNA targeting INHBE mRNA – a genetically validated target that provides a new approach for healthy, ...

Columnist Betty Vertin has learned that as a parent, she's finding the years with all her children, including those with DMD, too short.

Nonprofits that challenged to the Trump administration’s freezing of grant funds highlighted crisis for a small center in ...

Scientists at the University of Exeter have identified an unexpected potential contributor to human health and longevity: ...

usz.ch Background: Duchenne muscular dystrophy (DMD) leads to progressive impairment of ... dependence on care and respiratory impairment in patients with DMD from childhood to adult life is valuable ...

Shares of PTC Therapeutics, Inc. (NASDAQ:PTCT – Get Free Report) shot up 6.1% during trading on Monday after Cantor Fitzgerald raised their price target on the stock from $76.00 to $113.00. Cantor ...

It was the type of upsetting yet commonplace problem most parents face from time to time. Early last month, the Taylor family were on their way home from a meal out when Trey, 11, began feeling sick.