MDA Launches 38th Annual #Shamrocks Fundraising Campaign, Aims to Raise $8M

The Muscular Dystrophy Association (MDA) has launched its 38th annual #MDAShamrocks campaign — the largest St. Patrick’s fundraiser in the U.S. — aiming to raise money and awareness about muscular dystrophy and…

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…

Adults with Duchenne muscular dystrophy (DMD) should help younger generations through the difficult parts of growing up with a rare muscular disease. They need someone in a similar situation to look up to, relate to, and learn from. I recently met a high schooler who…

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, “…

For those of you who follow my column, I have some good news: Sammy Eikahldit is back. My alter ego, Sammy, first appeared in my column on true accessibility for all. He returned a couple months later when we discussed how to effect positive change. Sammy has been…

  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…