Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

WVE-N531, an oligonucleotide, elicited significant functional benefit and reversal of muscle damage in the Phase II ...

“Quantitative MRI is a noninvasive methodology that is sensitive and can detect subclinical disease progression in Duchenne muscular dystrophy,” Vandenborne said. “The qualitative MR ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death of a patient taking Sarepta’s Elevidys raises important safety questions.

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...

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 ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...