News

This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

An advisory committee of the European Medicines Agency (EMA) has issued another negative recommendation after re-examining a request to extend Raxone (idebenone) for use in treating Duchenne muscular dystrophy (DMD). The EMA’s Committee for Medicinal Products for Human Use (CHMP) issued its first negative opinion of the request in…

Ezutromid significantly reduced muscle damage in patients with Duchenne muscular dystrophy (DMD) after 24 weeks of treatment in a Phase 2 trial, Summit Therapeutics recently announced. DMD is a severe type of muscular dystrophy caused by the absence of dystrophin, a protein…

A dysfunctional cerebellum leads to implicit learning deficits in children with Duchenne muscular dystrophy (DMD), Italian researchers have suggested. Their study, “Implicit learning deficit in children with Duchenne muscular dystrophy: Evidence for a cerebellar cognitive impairment?” appeared in the journal PLoS ONE. DMD is an…

Researchers have found that cognitive function, including the ability to multitask and solve problems, may be impaired in boys with Duchenne muscular dystrophy (DMD) — even in those with normal and above average IQs. Their study, “Cognitive profile in Duchenne muscular dystrophy boys without intellectual disability: The role of executive functions,”…

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…