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

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

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

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new therapeutic option for patients with the rare disease.

It can worsen with age. However, its symptoms are less severe than those of Duchenne muscular dystrophy (DMD), which is more common. Becker muscular dystrophy (BMD) is a type of muscular ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.

This story is republished from STAT, the health and medicine news site that’s a partner to the Globe. Sign up for STAT’s free Morning Rounds newsletter here. Wave Life Sciences’ experimental ...