WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial ...

Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North ...

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseasesCapricor remains on track for the ...

As the diseases progress, patients can experience muscle wastage and loss, which can severely affect their movement ability and sometimes their ability to breathe. There are around 30 types of ...

Cite this: Association of Duchenne Muscular Dystrophy With Autism Spectrum Disorder - Medscape - Oct 01, 2005. Abstract and Introduction; ... Muscle Biopsy Dystrophin Deletion by PCR ...

Becker Muscular Dystrophy, like Duchenne Muscular Dystrophy (DMD), is a progressive X-linked neuromuscular disorder that results in significant skeletal and cardiac muscle deterioration over time.

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease.

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

Jasper Day, from Horsham, has Duchenne muscular dystrophy, a genetic disorder characterised by progressive muscle degeneration and weakness, as well as his twin sister Arabella.

To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally rare and has only emerged in the non-ambulatory patients," Sarepta's CEO ...