Ana Pena, PhD,  —

Two magnetic resonance measures — MRI and magnetic resonance spectroscopy (MRS) — of leg muscles are quality biomarkers that help to predict clinical milestones in Duchenne muscular dystrophy (DMD), including loss of walking ability, and may serve as outcomes measures in clinical trials, a natural history study reported. Its…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to losmapimod, an investigational oral therapy for people with facioscapulohumeral muscular dystrophy (FSHD). This designation offers Fulcrum Therapeutics financial support to develop this treatment, as well as a waiver from the FDA’s fees and…

The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root cause of muscle degeneration in facioscapulohumeral muscular dystrophy (FSHD), new research in patient-derived cells and in a mouse model suggests. This supports a way forward in developing the first…

Parent Project Muscular Dystrophy (PPMD) has awarded two grants, one to further development of a gene therapy to prevent heart failure linked to Duchenne (DMD) and Becker muscular dystrophy (BMD), and another to create better measures of treatment responses in DMD clinical trials. PPMD, a nonprofit organization…

Catabasis Pharmaceuticals and the Jain Foundation have started a preclinical research collaboration to study edasalonexent as an oral treatment candidate for dysferlinopathy, a group of muscle diseases that includes limb-girdle muscular dystrophy (LGMD) type 2B. Edasalonexent, formerly CAT-1004, is a small molecule designed…

Most parents of children with Duchenne muscular dystrophy and adults with this disease — who took part in an interview study arranged and owned by Parent Project Muscular Dystrophy (PPMD) — expressed “cautious optimism” about gene therapy as a potential DMD treatment, weighing potential benefits against risks and disease…

An adverse event report was erroneously submitted for a patient enrolled in Study-102 testing Sarepta Therapeutics’ micro-dystrophin gene therapy, an experimental treatment for Duchenne muscular dystrophy (DMD), the company announced. The board in charge of the study’s safety found no reason to stop the trial. According to…

The Muscular Dystrophy Association (MDA) awarded $389,000 in funding to a researcher from University of California, Los Angeles (UCLA) to support the development of a small molecule that holds promise for preventing muscle damage in Duchenne muscular dystrophy (DMD). The molecule raises levels of a…

Early trial data supports the potential of FibroGen‘s investigative treatment pamrevlumab to slow the decline in, or even improve, lung and heart function in non-ambulatory youth with Duchenne muscular dystrophy (DMD), as well as to preserve their upper limb performance and strength. These findings were highlighted…

Enrollment is progressing quickly in Catabasis Pharmaceuticals‘ pivotal Phase 3 PolarisDMD trial, testing its investigational small molecule therapy edasalonexent for Duchenne muscular dystrophy (DMD) — with sites in some countries already full, the company said. Screening is ongoing in the U.S., Canada, Australia, the U.K.,…