An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according to…
Discussion
This week, I found myself reflecting on how much my life has changed since I joined Shalom Medcare, a care transport and medical escort provider in Singapore that was founded in 2021. Working there has become a part of how I advocate for healthcare equality for those who live…
Discussion
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD), met its main goal, the therapy’s developer, Regenxbio, announced in a press release. Steve Pakola, MD, Regenxbio’s chief medical officer, said the data “support the potential of RGX-202…
Discussion
They say the third time is the charm. However, as a mom to three sons with Duchenne muscular dystrophy (DMD), I don’t find that to be the case. I share seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and…
Discussion
Bridgebio is providing $100,000 to the Muscular Dystrophy Association (MDA) to advance projects aimed at improving multidisciplinary care for people with limb-girdle muscular dystrophy (LGMD). The awards will go to initiatives at Stanford Health Care and the University of Minnesota that aim to make LGMD care more…
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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