An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting Friday to discuss SRP-9001 (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy (DMD) that’s up for possible approval. The meeting, which will be livestreamed on YouTube, is scheduled from 9 a.m. to…
Readers of my column are familiar with my Duchenne muscular dystrophy (DMD) story, which began with my diagnosis in February 1996. My older brother, Isaac, was diagnosed with DMD at birth in August 1991. My family’s DMD story, however, dates back four decades from then, to 1951,…
Years of living with facioscapulohumeral muscular dystrophy (FSHD) have taught me that it’s a balancing act on a high wire. It’s an everyday challenge to keep from falling off one way or another. Overdo it and you spend a day or two paying the price with…
In people with Becker muscular dystrophy (BMD), both an elevated creatine-to-creatinine ratio and lower myostatin muscle protein levels in the bloodstream are associated with worse motor performance, a study reported. These two potential biomarkers predicted current functional abilities when combined with age but not disease progression over four years. Both…
My seven kids are my kids. That’s it. They all have strengths as well as weaknesses, challenges, and issues they must overcome. They also all have moments when they argue, talk back, get in trouble at school, and are genuine pains in the rear. I cannot tell you how…
After two years of treatment with losmapimod, people in a clinical trial with facioscapulohumeral muscular dystrophy (FSHD) showed no upper limb function worsening. Leo Wang, MD, PhD, of the University of Washington, presented the findings at the American Academy of Neurology (AAN) annual meeting in the talk, “…
Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD) one year after being given the gene therapy SRP-9001 (delandistrogene moxeparvovec) continued for a second year, according to new clinical trial data presented at the recent American Academy of…
AOC 1044, an investigational exon 44-skipping therapy for Duchenne muscular dystrophy (DMD), has been granted fast track status by the U.S. Food and Drug Administration (FDA). The designation is intended to accelerate the therapy’s development and expedite its approval by providing more frequent meetings with the FDA and discussions…
It happened two weeks ago. Then it happened again yesterday. Is this my new normal? If so, what to do? Should I laugh? Should I cry? Is a little spilled milk worth getting upset? I’m an early riser, getting up each morning to say some prayers and read some meditations.
This month, I had the rare opportunity to meet Dr. Brenda Wong, a native Singaporean and a professor of pediatrics and neurology at the University of Massachusetts Chan Medical School, where she was the founding director of its Duchenne Program. My parents had heard Dr. Wong’s name since…
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