A rare and fatal form of muscular dystrophy has long posed a devastating diagnosis for children, primarily affecting boys.

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

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 ...

Acute myeloid leukemia (AML) can cause many different signs and symptoms. Some are more common with certain subtypes of AML. More often these symptoms are caused by something other than leukemia. But ...

The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra Bonner of the Center for Pediatric Neurosciences at Cleveland Clinic.

2 The current retrospective, US population-based, longitudinal study used surveillance data from the multi-site Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet ...

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 ...

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 ...

Duchenne muscular ... dystrophy. It is a progressive genetic disorder that primarily affects boys, leading to gradual muscle degeneration and loss of motor function. In addition to physical ...

Cas12Max-based gene editing therapy for Duchenne muscular dystrophy, one of the most severe forms of the inherited muscular dystrophies that affects primarily boys. Huidagene Therapeutics and ...

An oral presentation will focus on the treatment of Hemophilia A while new data in the poster session targets the treatment of Duchenne Muscular Dystrophy (DMD). Both presentations at ASGCT feature ...

Methods A custom solution-based target enrichment kit was designed to capture whole genomic regions of the dystrophin gene and other muscular-dystrophy-related genes. A multiplexing strategy, wherein ...