Alice Melão,  —

A new mouse model of Duchenne muscular dystrophy (DMD) can help researchers test potential gene editing therapies by showing real-time levels of the dystrophin protein in muscles and the heart, a study suggests. The new strategy is a sensitive,…

Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (DMD), real-world data show. The most recent results from the ongoing STRIDE registry — an observational study across more than 50 care centers in Europe and Israel — were presented at…

Experimental therapy ATL1102 improved upper limb strength and function in six boys with Duchenne muscular dystrophy (DMD) unable to walk, according to early results of an ongoing Phase 2 clinical trial. These results also indicate that the therapy has been well-tolerated with no safety concerns reported so far.

Cumberland Pharmaceuticals announced that it has been awarded a $1 million grant by the U.S. Food and Drug Administration (FDA) to support a Phase 2 clinical trial into oral ifetroban as potential treatment of heart disease associated with Duchenne muscular dystrophy (DMD). The award marks the first time…

The ongoing Phase 2 HOPE-2 study has been cleared to continue exploring the potential of CAP-1002 therapy in boys and young men with advanced Duchenne muscular dystrophy (DMD). An independent Data and Safety Monitoring Board (DSMB) made the decision after its thorough safety and futility review and the…

A signaling pathway determines whether muscle stem cells differentiate and mature into working muscle cells, an early study in mice found. Targeting this pathway and its two proteins — Fam3a and Stat3, which works to stimulate Fam3a — may treat diseases or illnesses of muscles, including muscular dystrophies. Getting these stem cells to…

Engineered small versions of dystrophin protein, known as microdystrophins, have shown the ability to enhance muscle strength and function in preclinical models of Duchenne muscular dystrophy (DMD). Researchers from the University of Washington have now developed enhanced versions of these microdystrophins that can overcome some of the…

Severe Duchenne muscular dystrophy (DMD) is linked to increased susceptibility to a rare and aggressive type of muscle cancer called rhabdomyosarcoma, a mouse study suggests. The finding was reported by researchers from Sanford Burnham Prebys Medical Discovery Institute in the study, “Muscle Stem Cells…

Early safety and efficacy data from a Phase 1 trial suggest that Taiho Pharmaceutical’s investigational compound TAS-205 may hold the potential to treat patients with Duchenne’s muscular dystrophy (DMD). The results were reported in the study, “A phase I…

Researchers at The University of Western Australia (UWA) have been awarded close to $490,000 from two organizations to explore the potential use of an amino acid called taurine as a treatment for Duchenne muscular dystrophy (DMD). Duchenne UK granted £273,648 (approximately $356,515) to support the study, while Save our Sons…