Brogidirsen (NS-089/NCNP-02), NS Pharma’s investigational therapy for people with Duchenne muscular dystrophy (DMD) who are amenable to exon 44 skipping, has been shown to sustain motor function and maintain a favorable safety profile over 3.5 years of treatment in an open-label extension study. The therapy is designed to…
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In the past couple months, I’ve had more medical challenges than usual. My right arm hasn’t been working well, leading to some additional struggles with activities of daily living. In June, what I thought was a bug bite appeared on the knuckle of my right middle finger. It would swell…
Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European…
Moms worry about their children. As a mother of seven — Lexi, 24; Max, 19; Chance, 18; Rowen, 16; Charlie, 14; Mary, 11; and Callie, 3 — I know firsthand what it’s like to be anxious and concerned about kids. My children’s ages span 20 years — another thing to…
Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada. Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S., the U.K.,…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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