News

People known as “consumer advocates” have joined top scientists since 2011 in helping to determine how funds appropriated by the U.S. Congress will be spent on Duchenne muscular dystrophy (DMD) research. Recently, Tiffany Cook and Clint M. Porter got their turn at patient representation. Cook and Porter’s influence derived…

Registration for the upcoming Parent Project Muscular Dystrophy (PPMD) Advocacy Conference is now open. During the conference, which will be held March 8–10 in Washington, D.C., participants will receive extensive training and support to empower and prepare them to advocate for Duchenne muscular dystrophy…

Oral treatment with TAS-205 is safe, and may delay muscle deterioration in patients with Duchenne muscular dystrophy (DMD), an early Phase 2 clinical trial suggests. The findings were detailed in a study, “…

  Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the world. The Vivli platform, which currently hosts information from more than 4,700 clinical…

The U.S. Food and Drug Administration (FDA) accepted NS Pharma‘s new drug application for viltolarsen, a potential treatment for Duchenne muscular dystrophy (DMD) patients who are amenable to exon 53 skipping, the company and its parent, Nippon Shinyaku, said. The companies expect a decision on approval between…

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

A new project called BIND — Brain Involvement  iN  Dystrophinopathies — seeks to better characterize how the brain is affected in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), and to find potential ways to treat neurological complications in people with those disorders. The European Union awarded…

Eighty delegates from 37 countries recently met in Athens for the 2019 Duchenne Patient Academy — a three-day series of master classes aimed at deepening global understanding of Duchenne muscular dystrophy (DMD) and coordinating advocacy strategy. The Dec. 6-8 conference, an annual event since its 2017 start, covered subjects…

Orsini Pharmaceutical Services is now providing Sarepta Therapeutics’ newly approved therapy Vyondys 53 (golodirsen) to treat people with Duchenne muscular dystrophy (DMD) responsive to exon 53 skipping…

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.