News

Rare pediatric disease designation granted to LAMA2-CMD therapy

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…

DMD treatment WVE-N531 boosts dystrophin: Interim trial data

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…

MDA Engage offers neuromuscular disease updates, connections

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…

Losmapimod fails to significantly improve FSHD outcomes: Study

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…

Atamyo, Dion Foundation partner to expand LGMD study into US

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…