News

New Drug Applications (NDA) for Marathon’s investigational drug deflazacort have been accepted for filing and granted Priority Review by the U.S. Food and Drug Administration (FDA). The NDAs, one for immediate-release tablet formulations and one for an oral suspension formulation, request that deflazacort be approved for the treatment of Duchenne muscular dystrophy…

The first patients have been dosed in PhaseBio Pharmaceuticals‘ part one of a Phase 2a clinical trial for PB1046, a once-weekly vasoactive intestinal peptide (VIP) receptor activator currently in development to treat cardiopulmonary disorders. Part one is designed to assess the effect of PB1046 in adult patients with stable heart failure with reduced ejection fraction…

Bamboo Therapeutics has been fully acquired by Pfizer, considerably expanding that company’s holdings in gene therapy. With the acquisition, Pfizer gains several potential therapeutic agents for rare diseases, including one for Duchenne muscular dystrophy (DMD), soon to enter clinical testing; and one Phase 1/2 clinical stage agent, a potential best-in-class recombinant Adeno-Associated Virus (rAAV)…

Researchers have developed a new gene therapy for muscle wasting conditions, and are now working to bring into clinical use through a newly formed biotech venture. The study describing the method, “Smad7 gene delivery prevents muscle wasting associated with cancer cachexia in mice,” published in the journal…

The Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF) are joining forces this summer to support efforts to find much-needed treatments, and possibly a cure, for muscular dystrophy (MD), amyotrophic lateral sclerosis (ALS) and other muscle-degenerative diseases. Members of…

PTC Therapeutics has provided global regulatory updates on its lead product candidate Translarna (ataluren) for treating nonsense mutation Duchenne muscular dystrophy (nmDMD) and nonsense mutation cystic fibrosis (nmCF). According to the National Institutes of Health Genetics Home Reference, a nonsense mutation is a change in one DNA base pair…

The Child Neurology Foundation (CNF) recently published a statement in the journal Neurology to address the role of neurologists in transferring pediatric patients into the adult healthcare system. The statement was published in July 27 issue under the title “The neurologist’s role in supporting transition to adult health care:…

Two British physiotherapists invited to speak at a July 20 event at the Houses of Parliament in London by Muscular Dystrophy UK, informed the Parliament and commissioners about a serious shortage throughout the U.K. of physiotherapy for people with muscle-wasting diseases such as muscular dystrophy. Specialist neuromuscular physiotherapists Gita Ramdharry and…

The protein product of a gene called AUF1 determines whether muscle stem cells maintain the ability to regenerate muscles lost by injury or as a result of aging or disease, according to a study conducted on mice by researchers at New-York University (NYU), Langone Medical Center and the University of Colorado at Boulder.

California Stem Cell Agency (CIRM) awarded a total of $2,150,400, to scientists at the University of California Los Angeles (UCLA), Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and the Center for Duchenne Muscular Dystrophy at UCLA, for a cutting-edge stem cell gene therapy research project that could produce a treatment for Duchenne muscular dystrophy (MD).