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

Posters presented at the 2025 Muscular Dystrophy Association ... health benefits for individuals with myotonic dystrophy, multisystemic characteristics of myotonic dystrophy often lead to ...

"Overexpressing MBNL led to the restoration of some heart functions and characteristics ... This work was funded by Muscular Dystrophy Association (grant #276796), National Institutes of Health ...

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.

Overexpressing MBNL led to the restoration of some heart functions and characteristics ... This work was funded by Muscular Dystrophy Association (grant #276796), National Institutes of Health ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a ... developed that replicates ...