Heart problems can progress quickly in people with myotonic dystrophy type 2 (MD2) or facioscapulohumeral muscular dystrophy type 1 (FSHD1), even in those showing no symptoms of cardiac disease, a study reported. “MD2 and FSHD1 patients should be carefully followed-up to identify early development of remodeling and potential risks for the development…
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Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
Significantly higher levels of sphingolipids — a type of fatty molecule involved in inflammation, scarring, and cell death — are evident in the muscles of people with Duchenne muscular dystrophy and a DMD mouse model than in those of their healthy counterparts, a study shows. Notably, suppressing sphingolipid production in…
Life expectancy has been increasing in patients with Duchenne muscular dystrophy (DMD), according to an analysis of some 40 years of data, spanning births from before 1970 to the late 2000s. In fact, the median life expectancy of males with DMD born in the late 1990s advanced to more than…
A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy CAP-1002 as a treatment for Duchenne muscular dystrophy (DMD), is expected to launch soon. Capricor Therapeutics, the company developing CAP-1002, announced that the U.S. Food and Drug Administration…
A protein called GCN5 is vital for normal muscle health and helps to coordinate the expression of several important muscle proteins, including dystrophin, according to a study done in mice. The results suggest that targeting GCN5 may be a useful therapeutic avenue in diseases of the muscles, such as…
The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical trial evaluating DYNE-251, an experimental exon-skipping therapy for Duchenne muscular dystrophy (DMD). Dyne now hopes to have that Phase 1/2 trial underway by June, the company said in a …
Cognitive problems seen in some Duchenne muscular dystrophy (DMD) patients may be associated with a shift toward the amyloidogenic pathway in memory-specific brain regions, according to a study in mouse models of the disease. The amyloidogenic pathway is a signaling cascade that leads to the formation of beta-amyloid, the…
The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in the U.S. evaluating the safety and efficacy of RGX-202, its experimental gene therapy for Duchenne muscular dystrophy (DMD). The study, called AFFINITY DUCHENNE and anticipated to start in…
Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked improvement in motor abilities after being treated with Sarepta Therapeutics‘ experimental gene therapy SRP-9001, new top-line data show. “We are delighted to report positive results for Part 2 of…
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