Margarida Azevedo, MSc,  —

The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics and Nationwide Children’s Hospital recently announced. Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a…

The nonprofit CureDuchenne launched a new resource for the Duchenne muscular dystrophy (DMD) community called the CureDuchenne Cares Virtual Durable Medical Equipment (DME) Expo. CureDuchenne was founded in 2003 to help save the lives of those with DMD. Through CureDuchenne Cares, the organization has brought innovative…

Casey’s General Stores  customers contributed $1.35 million in August to support the Muscular Dystrophy Association‘s 2017 Summer Camp, where children living with MD enjoy a week of fun and make new friends at no cost to their families. Since the fundraising campaign was launched in 2006, $9 million has been…

Two non-profits that work on standards that can facilitate drug development have created a guide to help those doing Duchenne’s muscular dystrophy research. The two are Critical Path Institute (C-Path) and the Clinical Data Interchange Standards Consortium (CDISC). The resource they created is the Duchenne Muscular Dystrophy Therapeutic Area User…

Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial. The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint…

Parent Project Muscular Dystrophy (PPMD), one of the largest nonprofit organizations in the U.S. working to find a cure for Duchenne muscular dystrophy (DMD), recently announced that it will continue to support a grant for the Certified Duchenne Care Center (CDCC) Program. PPMD invests in research, advocacy, and initiatives to…

The newly founded Collaborative Trajectory Analysis Project (cTAP) coalition of stakeholders has invited Summit Therapeutics, a company specialized in the discovery and development of drugs targeting Duchenne muscular dystrophy (DMD), to support the development of new therapeutic products aiming to treat DMD, such as utrophin modulators. cTAP…

The Muscular Dystrophy Association (MDA) has launched its 2017 “Live Unlimited” awareness and fundraising campaign. Running until Oct. 11, it targets families affected by muscular dystrophy (MD) and other neuromuscular diseases, and aims to help patients pursue their personal and professional goals. This year’s campaign features inspiring examples:…

The nonprofit group CureDuchenne won $50,000 at a recent Shark Tank-like competition in Irvine, California, to pitch scientific projects. The “RARE Battle of the Brains” competition, held Sept. 14, was organized by Scientist.com, a marketplace for scientific services, and Global Genes, a rare disease patient advocacy group. CureDuchenne won the…

Phrixus Pharmaceuticals and several Duchenne muscular dystrophy (DMD) organizations will jointly conduct the first open-label trial of Poloxamer-188 NF, the active ingredient in Carmeseal-MD, to treat DMD in patients who can no longer walk. The trial, to take place at Cincinnati Children’s Hospital, will evaluate the drug’s performance on respiratory…