The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as…
Articles by Steve Bryson, PhD
A young man with Duchenne muscular dystrophy (Â鶹Éç) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has…
Preventive treatment with standard heart medications — when given before the onset of cardiac troubles — extends survival among males…
Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with…
The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle…
A noninvasive quantitative MRI, or qMRI, was found to detect early muscle abnormalities among people with limb-girdle muscular dystrophy…
Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase…
One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults…
After three years of treatment with Capricor Therapeutics’ experimental cell therapy CAP-1002, people with Duchenne muscular dystrophy (Â鶹Éç)…
A $250,000 award from the Parent Project Muscular Dystrophy (PPMD) will support the establishment of Baby Duchenne, a collaborative…