Janet Stewart, MSc,  —

CureDuchenne, a nonprofit advocacy group that supports and promotes research and better care and for people with Duchenne muscular dystrophy (DMD), has announced the launch of a three-year partnership with Baebies in which the company will add DMD to its expanded newborn screening service. The goal is to make…

The Pacific Northwest chapter of the FSH Society is holding its first-ever “Walk & Roll to Cure FSHD” event Sept. 22 in Puyallup, Washington, a small city about 35 miles south of Seattle. FSH Society holds these events around the U.S., with the goal of raising funds for its work…

Acceleron’s ACE-083, for the treatment of patients with facioscapulohumeral muscular dystrophy (FSHD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA), the company announced. FSDH affects about 20,000 people in the U.S. It is a rare genetic muscle disorder for which there…

PTC Therapeutics announced the start of its fourth annual STRIVE awards program for Duchenne muscular dystrophy (DMD). STRIVE — which stands for Strategies to Realize Innovation, Vision and Empowerment — supports programs run by nonprofit patient advocacy groups that aim to increase disease diagnosis, awareness and education, and train…

Expansion Therapeutics has raised $55.3 million in Series A financing to advance its portfolio of drugs targeting several genetic disorders, including myotonic dystrophy type 1 (DM1), the leading cause of adult onset muscular dystrophy. Toxic levels of RNA are thought to…

Researchers tracked the way adult skeletal muscle stem cells (MuSCs) multiply in response to aging or injury. The study has implications for treatments in diseases of muscle tissues, such as muscular dystrophies. The study, “Muscle Stem Cells Exhibit Distinct Clonal Dynamics in Response to Tissue Repair and Homeostatic…

Summit Therapeutics announced the completion of a 24-week dosing period of ezutromid, its investigational therapy for patients with Duchenne muscular dystrophy (DMD). Summit expects to report the results of this initial dosing period of its Phase 2 trial during the first quarter of 2018. The Phase 2 PhaseOut DMD…

Exonics Therapeutics has secured $40 million in financing from The Column Group (TCG) — a science-driven venture capital firm — to develop its lead gene editing program in Duchenne muscular dystrophy (DMD). Exonics, based in Cambridge, Massachusetts, is working to develop a safe, one-time gene editing treatment that would provide lifelong…

Results from the HOPE-1 trial of Capricor Therapeutics’ cell-based therapy CAP-1002 to treat boys and men at advanced stages of Duchenne muscular dystrophy (DMD) will be released at a late-breaking session at the American Heart Association Scientific Sessions 2017. In a session…

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory…