Seeking to build on the positive results seen among boys with Duchenne muscular dystrophy (DMD) treated with its experimental exon-skipping therapy zeleciment rostudirsen — z-rostudirsen, formerly known as DYNE-251 — in an early clinical trial, Dyne Therapeutics has launched a global confirmatory study, dubbed FORZETTO. This new Phase 3…
Discussion
Having a rare disease is not an easy life. I have limb-girdle muscular dystrophy, so in addition to the physical decline of my body, I also need to be hyperaware of my mental health. I don’t think humans were hardwired to adjust to life on their own with a…
Discussion
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…
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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