News

MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than…

The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and age at symptom onset, according to the findings of a four-year study, but the researchers nonetheless reported “notable variability” with patients’ general decline. The study, by researchers in the Netherlands, focused on changes over…

The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines Agency — again maintained its recommendation not to renew conditional marketing authorization for Translarna (ataluren), a treatment for Duchenne muscular dystrophy (DMD) caused by nonsense mutations. The European Commission, which has final say…

Treatment with antimiRs, an RNA-based therapy approach, was able to correct molecular defects in a cell model of myotonic dystrophy type 1 (DM1) — a form of muscular dystrophy — according to the results of a new study. The researchers called their work “a significant step forward,” and noted that…

A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystrophy (OPMD) treated in a Phase 1b/2a clinical trial, according to an update from the therapy’s developer, Benitec Biopharma. Trial findings to date, which include reasonable…

Capricor Therapeutics has started its rolling submission seeking U.S. approval of deramiocel, a cell therapy candidate to treat cardiomyopathy, a disease of the heart muscle, in people with Duchenne muscular dystrophy (DMD), the company announced. Rolling submission means that Capricor will be submitting parts of the process to…

The Critical Path Institute (C-Path) is launching a task force to advance the development of therapies for limb-girdle muscular dystrophy (LGMD) and will lead the efforts of its members to find a new treatment to, among other goals, slow or stop the progression of the muscle-wasting disease. Formed…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to MDL-101, an epigenetic editing therapy that Modalis Therapeutics is developing for LAMA2-related congenital muscular dystrophy (LAMA2-CMD) — a genetic condition that’s characterized by muscle wasting evident at birth or shortly thereafter. This FDA status…

Treatment with exon 53-skipping therapy WVE-N531 led to significant increases in muscle dystrophin levels along with signs of improved muscle health and regeneration in boys with Duchenne muscular dystrophy (DMD). That’s according to new six-month interim data from the Phase 1b/2a FORWARD-53 trial (NCT04906460), which also found…

Capricor Therapeutics will soon begin filing an application seeking U.S. approval of its cell therapy deramiocel for cardiomyopathy, a disease of the heart’s muscle, in people with Duchenne muscular dystrophy (DMD). Following recent meetings with the U.S. Food and Drug Administration (FDA), Capricor will file a rolling…