News

The first Duchenne muscular dystrophy (DMD) patient has received systemic microdystrophin gene therapy as part of a Phase 1/2 clinical trial at Nationwide Children’s Hospital in Columbus, Ohio. The trial (NCT03375164), currently recruiting participants, will test the safety and efficacy of a single dose of intravenous microdystrophin gene therapy…

A global Phase 3 trial of givinostat — an investigational treatment aiming to boost the muscle regeneration capacity in Duchenne muscular dystrophy patients — is recruiting participants across North America and Europe. The trial is looking to recruit boys, older than six years, who are still ambulatory and on a stable…

Capricor Therapeutics is expanding its Duchenne muscular dystrophy (DMD) and cardiac therapy programs using cardiosphere-derived cells (CDCs) by adding seven new patent applications related to the technology. The new applications were from Capricor’s existing exclusive license agreements with Cedars-Sinai Medical Center, and are aimed at expanding Capricor’s cellular and…

The U.S. Food and Drug Administration (FDA) recently granted the gene therapy BB-301 orphan drug status for the treatment of oculopharyngeal muscular dystrophy (OPMD), according to Benitec Biopharma. OPMD is caused by a mutation of the poly(A)-binding protein nuclear 1 (PABPN1) gene. The resulting faulty protein can form…

Seven pharmaceutical companies are banding together to sponsor Project Hercules — an initiative that addresses evidence generation for Duchenne muscular dystrophy (DMD) treatments. The project, led by the non-profit organization Duchenne UK, aims to simplify the way evidence is generated for health technology assessments by authorities such as Great Britain’s National…

Researchers have found that Duchenne muscular dystrophy patients usually have simultaneous neurological disorders or abnormalities, including epilepsy. Particularly, those who had epilepsy also had the neurodevelopmental condition attention deficit hyperactivity disorder (ADHD), or the neuropsychiatric conditions obsessive-compulsive disorder, anxiety or sleep disorders. Since the research, published in the European…

Clinical data from a Phase 3 trial revealed that Kyndrisa (drisapersen) could not significantly improve the walking ability of boys with Duchenne muscular dystrophy (DMD) due to mutations amenable with exon 51 skipping. Still, additional analysis of the DEMAND III trial (NCT01254019) showed this therapy may still…

Researchers have succeeded in growing working muscle tissue from a skin sample — a feat that until recently was viewed as science fiction. Although the lab-grown muscle was not as strong as those in the body, the advance opens up for new approaches to tissue regeneration in patients with muscular dystrophy…

Acceleron Pharma’s treatment for a muscular dystrophy affecting the face and other muscles increased the muscle mass of those with the disorder, according to a Phase 2 clinical trial. The results covered the first stage of the trial, which is assessing ACE-083’s3 ability to treat  facioscapulohumeral dystrophy, or FSHD. FSHD…

Apple Tree Partners has invested $40 million in Stoke Therapeutics to come up with gene-expression treatments for muscular dystrophy and other genetic disorders. Stoke aims to develop antisense oligonucleotides to promote targeted augmentation of nuclear gene output (TANGO). The goal is to reverse the features of rare diseases…