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…
The U.S. Food and Drug Administration has granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping Duchenne muscular dystrophy (DMD) treatment from NS Pharma. Treatments granted this designation are aimed at serious or life-threatening diseases affecting children and fewer than 200,000 U.S. patients. “We are grateful for this…
Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S. a clinical trial of ATA-200, a potential gene therapy for limb-girdle muscular dystrophy (LGMD) type 2C/R5 — dubbed LGMD2C/R5. The Phase 1b study (NCT05973630), designed to evaluate the…
Up to a year of treatment with DYNE-251, Dyne Therapeutics’ investigational exon 51-skipping therapy, led to improvements in motor function for boys with Duchenne muscular dystrophy (DMD), according to a clinical trial update. Based on these positive data from the Phase 1/2 DELIVER clinical trial (NCT05524883), Dyne…
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