Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.

gstt.sthames.nhs.uk Although bladder function is thought to be unaffected in Duchenne muscular dystrophy, 46/88 boys interviewed had urinary problems. Nine underwent video urodynamics, showing in ...

Objective The relationship between functional dependence and quality of life (QOL) in Duchenne muscular dystrophy (DMD) patients and burden and QOL in caregivers is not clear. This study investigated ...

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

Satellos said that it will be advancing its DMD small molecule to a Phase II trial, following the Phase I data.