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 ...

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

"I'm so happy because I thought he wasn't going to make it," Taevion Norris' mother said at the graduation ceremony.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...

Dr. Binks brings 25 years of experience in clinical development and research, particularly in rare diseases and Duchenne muscular dystrophy (DMD). He has previously held key positions at Pfizer ...

While genetic testing could not confirm Duchenne muscular dystrophy, a muscle biopsy at UCLA did. "He could see that he was declining, and that it was getting worse," said Stephanie. Pediatric ...

“For nearly a decade, Sarepta has been a leader in advancing the treatment of Duchenne muscular dystrophy through innovative treatment options. With the approval of ELEVIDYS in Japan, children who are ...

Earlier this year, Una Ennis took to social media with a heartfelt plea for funds so she could access specialist treatment abroad, following her seven-year-old son Archie’s muscular dystrophy ...

It slows progress of Duchenne muscular dystrophy (DMD) but patients in Scotland have not been able to get the drug despite manufacturers offering it for free on an early access programme.