News

Akashi Therapeutics Inc. a biopharmaceutical company focused on developing treatments for patients with Duchenne muscular dystrophy (DMD) recently announced encouraging interim results of its ongoing Phase 1b/2a clinical trial of HT-100 (delayed-release halofuginone) an orally available small molecule to reduce inflammation and fibrosis while promoting the regeneration of…

Researchers at UCL Institute of Child Health in the United Kingdom reported new insights into muscle stem cell transplantation therapy in muscular dystrophy mouse models. The study was recently published in the journal Skeletal Muscle and is entitled “The effect of the muscle…

Patients with facial paralysis as a result of muscular dystrophy may benefit from new research conducted at Johns Hopkins University School of Medicine. Kofi Boahene, MD, a facial plastic and reconstructive surgeon in the Department of Otolaryngology-Head and Neck Surgery at Hopkins, led a study that found hyaluronic…

The US Food and Drug Administration (FDA) this week released a new draft guidance document intended to clarify the development process for new Duchenne Muscular Dystrophy drug treatments. The purpose of this guidance is to assist sponsors in clinical development of drugs treatment of Duchenne muscular dystrophy (DMD) and…

BioBlast Pharma Ltd., a biotechnology company focused on rare genetic diseases, recently announced the start of a Phase 3 pivotal trial (NCT02328482) in the United States and Canada for its product Cabaletta (trehalose) for oculopharyngeal muscular dystrophy (OPMD). OPMD is a rare inherited neuromuscular disease characterized by muscle…

Massachusetts-based charity the FSH Society, dedicated to shedding light on the acioscapulohumeral muscular dystrophy (FSHD) — a little-known variant of the incurable disorder, has been awarded its seventh consecutive Charity Navigator four-star rating. Only two percent of the charities rated by the Charity Navigator — an independent nonprofit…

A study recently published in the journal PNAS revealed new insights into a gene called Smchd1 that is involved in muscular dystrophy development. The study is entitled “Genome-wide binding and mechanistic analyses of Smchd1-mediated epigenetic regulation” and was conducted by a team led by researchers…

A lightweight robotic glove developed by engineers at the Wyss Institute for Biologically Inspired Engineering and Harvard John A. Paulson School of Engineering and Applied Sciences (SEAS) in Boston and Cambridge, Massachusetts is bringing the ultimate goal of providing muscle and neurologically impaired patients with the ability to…

Non-profit organization Parent Project Muscular Dystrophy (PPMD) has nominated Duke Children’s Hospital & Health Center as a Certified Duchenne Care Center, a designation that confirms the center’s quality in the treatment of Duchenne muscular dystrophy. Duke Children’s is now the ninth center in the country to be…

A novel study from Max Planck Institute for Heart and Lung Research investigators revealed a novel protein, Prmt5, as a crucial player in the regulation of muscle stem cells during muscle regeneration. The study entitled “Prmt5 is a regulator of muscle stem cell expansion in adult mice”…