Margarida Azevedo, MSc,  —

Parent Project Muscular Dystrophy (PPMD) has added a pre-conference workshop to this year’s 23rd Annual Connect Conference, inviting international medical experts to discuss inflammation and immunity in Duchenne muscular dystrophy (DMD). The conference, set for June 29-July 2, is the largest international conference dedicated entirely to DMD. Each year, the event…

Eloxx Pharmaceuticals received a $6 million investment from industry partners to advance a novel therapy for genetic diseases caused by nonsense mutations, including Duchenne muscular dystrophy (DMD), raising the total round of financing to $30 million. The investment will be used to advance the development of ELX-02, the…

Raxone (idebenone)  has been granted temporary scientific approval to be used to treat certain patients with Duchenne muscular dystrophy (DMD) in the United Kingdom (UK). The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) granted Santhera Pharmaceuticals‘ drug candidate a positive Early Access to Medicines Scheme (EAMS)…

Sarepta Therpeutics and Genethon have signed a research collaboration agreement to jointly develop a gene therapy for Duchenne muscular dystrophy (DMD). Created by the AFM-Telethon, the French Muscular Dystrophy Association, Genethon is a nonprofit organization that specializes in the discovery and development of drugs for orphan genetic…

On June 20, the Massachusetts-based FSH Society and the FSHD Champions ask everyone to join World FSHD Day to raise awareness for facioscapulohumeral muscular dystrophy (FSHD). FSHD patients, their families and their supporters as well as advocacy organizations will come together to urge a cure for one of…

Advocates from Save Our Sons Duchenne Foundation (SOS), in partnership with Parent Project Muscular Dystrophy (PPMD), have launched Australia’s first patient-led registry to increase clinical trial recruitment in Duchenne muscular dystrophy (DMD). The initiative was inspired by PPMD’s DuchenneConnect patient registry, which since 2007 has provided data…

Sarepta Therapeutics is recruiting up to 99 boys with Duchenne muscular dystrophy (DMD) to test two new compounds, SRP-4045 and SRP-4053, in a Phase 3 clinical trial. The ESSENCE study is a double-blind, placebo-controlled, multi-center Phase 3 clinical trial (NCT02500381) with an open-label extension period, evaluating the two drugs’…

A gene therapy treatment may restore the genetic mutations of myotubular myopathy, a type of muscular dystrophy that affects the skeletal muscles. In a previous study, a team of researchers at the University of Washington School of…

A new clinical-stage biotech, Myonexus Therapeutics, is developing gene therapies for limb-girdle muscular dystrophies (LGMDs). Launch plans include the accelerated development of five investigational gene therapies that have the potential to become the first standard-of-care therapies for LGMDs. Myonexus’ pipeline includes three clinical-stage gene therapy programs (LGMD2E, LGMD2D, and…

The U.S. Food and Drug Administration (FDA) scheduled a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting with PTC Therapeutics to review the company’s new drug application (NDA) for Translarna (ataluren) to treat patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The meeting will take place on Sept. 28, the company…