Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their ability to swallow after receiving the gene therapy candidate BB-301 in an ongoing clinical trial. That’s according to an update from therapy developer Benitec Biopharma, which announced new…
Discussion
On Jan. 31, I visited the former Supreme Court Foyer at the National Gallery Singapore (NGS) for the final Artsplaining session of Light to Night Singapore 2026. NGS is a leading visual arts museum with the largest collection of Southeast Asian modern art. Artsplaining is its program designed…
Dosing has begun in a Phase 2 clinical trial testing the oral therapy SAT-3247 in boys with Duchenne muscular dystrophy (DMD). The study, dubbed BASECAMP (NCT07287189), aims to enroll 51 boys with DMD aged 7-9 who can walk. Patients who have received treatments such as exon skippers,…
Discussion
I was born in March 1988 with the umbilical cord wrapped around my ankles three times. My heart rate dropped to zero, and the doctors had to do an emergency cesarean section to save me. I still reached all my milestones like a normal baby. When I was 6 years…
Discussion
It’s been a busy couple of weeks. There have been lots of appointments and activities, and I’ve been busy running errands around town. I live in a rural community in south central Nebraska. It’s small enough that, no matter where I go, I run into someone I know. I also…
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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