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…
The Muscular Dystrophy Association (MDA) is providing neuromuscular disease (NMD) patients and their families with the opportunity to meet experts and other people on similar journeys through its Engage Symposiums, free in-person events that include disease-specific sessions as well as talks focusing on NMDs in general. The association…
SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types.
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD), according to top-line data from the REACH Phase 3 trial. Participants given losmapimod did see improvements over 48…
Get regular updates to your inbox.