30 days of MD: Light It Up Green for MD

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Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Many people are inspired by a son or brother with Duchenne muscular dystrophy (DMD). When it comes to Danielle Edwards, she’s literally made the disease her career. Edwards, 26, is the community engagement manager at the Jett Foundation, a Massachusetts charity that helps families deal with the emotional and…

Let’s really get serious about what accessibility is. The website Disabled World calls it the “‘ability to access’ the functionality, and possible benefit, of some system or entity.” In short, the more people who can use the product, service, or venue, the more accessible it is. This is a…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Most parents of children with Duchenne muscular dystrophy and adults with this disease — who took part in an interview study arranged and owned by Parent Project Muscular Dystrophy (PPMD) — expressed “cautious optimism” about gene therapy as a potential DMD treatment, weighing potential benefits against risks and disease…

Last week, I wrote an open letter to parents of kids with Duchenne muscular dystrophy. Now I want to offer some words of advice to those kids. Your parents love you and will sacrifice a lot for your well-being. That means you have an even greater responsibility as a…

The nonprofit CureDuchenne has chosen veteran advocacy leader Erin Frey to become its first director of Duchenne muscular dystrophy (DMD) advancement efforts. In her official capacity as senior director of advocacy, Frey is charged with establishing relationships that promote CureDuchenne’s outreach, and connect Duchenne families and other…

The U.S Food and Drug Administration (FDA) has rejected Sarepta Therapeutic’s application seeking accelerated approval of golodirsen (SRP-4053) for Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. Golodirsen (SRP-4053) was placed under priority review by the FDA in February 2019. The FDA’s complete response…