The U.S. Food and Drug Administration (FDA) has placed a hold on clinical testing of ENTR-601-44, Entrada Therapeutics‘ investigational therapy for people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 44 skipping. The decision was in response to the company’s investigational new drug (IND) application,…
On Nov. 27, I attended a live performance by ART:DIS musician Sky Shen, an old friend from the Muscular Dystrophy Association (Singapore) (MDAS). The show featured his recent debut EP, “Wheel You Love Me?” — a collection of songs he wrote about his experiences with…
Wave Life Sciences‘ experimental exon-skipping therapy WVE-N531 appears to be working as expected, according to early data from a small clinical trial of the therapy in a first group of three boys with Duchenne muscular dystrophy (DMD). “These data provide early evidence that WVE-N531 is … leading to…
It’s hard to believe: This is the 100th column I’ve written for Muscular Dystrophy News Today. It will also be my last for the near future. “Hawk’s-Eye View” has been a part of me for the past three years, as I graduated college, completed a five-month internship at…
Bend your mind. When we discovered our middle son, who has Duchenne muscular dystrophy (DMD), was on the autism spectrum, we helped him adjust to unexpected changes by using the words “bend your mind” as a cue. We haven’t used those words with him for years, but as…
Treatment with AOC 1001 eased myotonia, when muscles are unable to relax after a contraction, in adults with myotonic dystrophy type 1 (DM1), according to early data from the Phase 1/2 MARINA clinical trial. MARINA data also showed — in all treated participants — AOC 1001 was successfully delivered…
PGN-EDODM1, a therapy candidate for myotonic dystrophy type 1 (DM1), showed a favorable safety profile in preclinical studies, including those in non-human primates, PepGen, the company developing the treatment, announced. This work is expected to support a request to initiate a single ascending dose clinical trial of PGN-EDODM1…
The lights shone brightly, illuminating dancers dressed in red on the stage below. The rest of the theater was dark, save for an iPad glowing toward the back of the auditorium. I sat in the dark hall on a cold Saturday night in the small Nebraska town I call home.
The first patients have consented to share their clinical data with the Electronic Health Record (EHR) Study, according to an announcement from Parent Project Muscular Dystrophy (PPMD). The study’s goal is to help clinicians and researchers improve care and learn more about Duchenne and Becker muscular dystrophy. The…
Sarepta Therapeutics‘ investigational GALGT2 gene therapy helped stabilize muscle function when delivered at a higher dose to a boy with Duchenne muscular dystrophy (DMD), data from a Phase 1/2a clinical trial show. The other trial participant, a boy with more severe disease, had a slight lung function improvement…
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