Treatment with vamorolone continued to show a good safety profile and sustained efficacy across functional assessments after almost one year in boys with Duchenne muscular dystrophy (DMD), according to 48-week results of the Phase 2b VISION-DMD clinical trial. Data also showed no loss of efficacy in boys who switched…
Author and advocate Elijah J. Stacy is using what he has learned through adversity in battling Duchenne muscular dystrophy (DMD) to help others overcome their own challenges. DMD is a progressive muscle wasting disease that slowly robs a person’s muscle strength. In Stacy’s case, he lost the ability to…
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people…
Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).
The seventh annual Napa in Newport wine auction, an event that brings together acclaimed Napa Valley vintners, raised more than $1.5 million for CureDuchenne. The nonprofit will use those proceeds to support people with Duchenne muscular dystrophy (DMD), the most common form of MD, and to find…
A Phase 1 trial of SRP-9001, Sarepta Therapeutics‘ investigational gene therapy, is recruiting 3-year-old boys with Duchenne muscular dystrophy (DMD) who are able to walk for a new patient group in California, according to a press release from the Muscular Dystrophy Association. This group, being enrolled at the…
Upper limb strength, function, and muscle fat fraction continuously worsen over time in Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 53 skipping, according to three-year data from a natural history study. Notably, this progression of upper limb involvement was assessed reliably with strength, function, and muscle…
The 2021 holiday retail fundraising program to support the Muscular Dystrophy Association (MDA)Â recently opened. The multiple retailers involved in this year’s campaign hope to raise at least $500,000 to support research and care for people with muscular dystrophy, amyotrophic lateral sclerosis (ALS), and related neuromuscular diseases.
A Phase 1 clinical trial evaluating Edgewise Therapeutics’ experimental oral therapy EDG-5506 in healthy volunteers and men with Becker muscular dystrophy (BMD) has dosed the first BMD patients. The study (NCT04585464) is currently recruiting up to eight men, ages 18 to 55, with BMD at its…
Atamyo Therapeutics is seeking approvals in Europe to launch its first trial testing ATA-100, its investigational gene therapy for a type of limb-girdle muscular dystrophy (LGMD). The company, a spin-off from Genethon, a Paris-based genetics research laboratory, has submitted an application asking three European…
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