The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European Union be expanded to include people with Duchenne muscular dystrophy (DMD) as young as 2. The CHMP is an arm of the European Medicines Agency (EMA) tasked with reviewing…
Discussion
Starting daily corticosteroids earlier in young children with Duchenne muscular dystrophy (DMD) helps them move better, walk or run faster, and keep their muscles stronger than delaying treatment by about one year, underscoring the importance of early diagnosis for preserving motor function. These are findings from a Phase 3…
Discussion
Last Saturday, I lost a dear friend of mine, Timothy Chan, who had Duchenne muscular dystrophy. He passed away in the hospital in the morning, days after he had been admitted with a respiratory illness. The unexpected news from his parents shocked me, as I had caught up with…
Discussion
Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of clinics dedicated to improving care for people affected by limb-girdle muscular dystrophy (LGMD). “LGMD has historically faced significant barriers to therapeutic development due to fragmented care infrastructure and extremely…
Discussion
Everyone recognizes the blue and white signs featuring a figure in a wheelchair that adorn certain parking spots. I call him the wheelchair guy, and he allows me to park close to public building entrances. The sign is an international symbol of disability. Of course, many disabled people are able…
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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