Researchers at the University of Oxford in the United Kingdom recently published in the journal Experimental Physiology a review on the recent progress in genetic based therapies for Duchenne muscular dystrophy (DMD). The study is entitled “Advances in genetic therapeutic strategies for Duchenne…
Myotonic muscular dystrophy type 1 is a chronic and slowly progressing genetic disease affecting many organs. It is the most frequent form of muscular dystrophy and, as the name implies, is characterized by wasting of the muscles throughout the body. Nevertheless, the genetic defect on the myotonic dystrophy protein kinase also…
Massachusetts based Catabasis Pharmaceuticals, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on their proprietary pathway pharmacology technology platform, recently announced that they have begun treating patients in their MoveDMD trial, a Phase 1/2 trial of CAT- 1004 for the treatment of Duchenne…
Grace Century’s bio banking project, Provia Laboratories, LLC, a healthcare services company specializing in high-quality bio-banking (the collection, transport, processing and cryogenic storage of biological specimens), recently reported 47% quarterly growth in enrollments for their Store-a-Tooth™ cryogenic storage service of dental stem cells. Store-A-Tooth is committed to offering…
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The US Food and Drug Administration (FDA) this week released a new draft guidance document intended to clarify the development process for new Duchenne Muscular Dystrophy drug treatments.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…
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