Charles Moore,  —

Summit Therapeutics announced that it has received a Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) for its lead drug candidate, ezutromid, as a treatment for Duchenne muscular dystrophy (DMD). The FDA defines Rare Pediatric Diseases as disorders that primarily affect people from birth to age…

The U.S. Food and Drug Administration has granted accelerated approval to Exondys 51 (eteplirsen), by Sarepta Therapeutics, making it the first drug approved to treat Duchenne muscular dystrophy (DMD) patients. Specifically, this treatment is for DMD patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.

Australia’s drug regulatory agency, the Therapeutic Goods Administration (TGA), has granted  Santhera Pharmaceuticals’ lead product Raxone (idebenone) orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). Raxone (sold in some markets under the trade name Catena) has already received a similar designation from European, Swiss, and…

 PTC Therapeutics marked Sept. 7 — World Duchenne Awareness Day — by announcing the four winner of the company’s 2016 global STRIVE Awards (Strategies to Realize Innovation, Vision and Empowerment) program. STRIVE awards are granted in recognition of extraordinary excellence and innovation by nonprofit organizations that serve rare disease communities, such as…

CureDuchenne, the Duchenne muscular dystrophy nonprofit  group engaged in disease advocacy and research funding,  announced on Sept. 7 — World Duchenne Awareness Day 2016 — that it has received a coveted STRIVE Award in recognition of its efforts. The awards were created by PTC Therapeutics to recognize, support, and honor…

Summit Therapeutics reports that a new formulation of ezutromid (called F6), its Duchenne muscular dystrophy (DMD) treatment candidate, achieved a greater than six-fold increase in maximum plasma levels in DMD patients participating in a Phase 1 clinical trial, compared to results achieved with its current formulation (F3), and at a lower…

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…

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…

Santhera Pharmaceuticals reports that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for its drug Raxone (idebenone;  U.S. brand name, Catena) as a treatment for Duchenne muscular dystrophy (DMD) in patients with respiratory function decline and who are not under glucocorticoid therapy. The EMA validation confirms…

Northbrook, Illinois-based Marathon Pharmaceuticals reports it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the corticosteroid drug deflazacort as a treatment for Duchenne muscular dystrophy (DMD). There is currently no FDA-approved treatment or cure for DMD, the most common and severe form…