The investigational gene therapy GALGT2 for Duchenne muscular dystrophy (DMD) was found to be safe in a preclinical study conducted at the Research Institute at the Nationwide Children’s Hospital, the researchers said. The study was funded by a $100,000 grant awarded by Parent Project Muscular…
College is a busy time in my life. I can easily overbook my schedule and have little time to do homework. I’ve resisted the temptation to stay up late on many occasions. I know the power of sleep. Sleeping about nine hours a night gives me a chance…
A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and…
First, a thank-you As I begin my second month as a columnist at Muscular Dystrophy News Today, I’m excited to be hearing from you about the columns. I’m on Twitter (@ralphyaniz) and always want to delve deeper. I also love some of the ideas I’m getting. As I…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
Merosin-deficient muscular dystrophy, a type of congenital MD, might be diagnosed before birth using immunohistochemistry, an easy and quick lab test that looks for specific markers in cells from a mother’s placenta, according to a small study of pregnant women whose children are at risk for this disease. A misdiagnosis, however,…
My life with mitochondrial myopathy has been a roller coaster, but my dog Andre was always there to brighten my world. After days spent healing in a hospital room, I could look forward to coming home to my beloved Andre. If I was suffering through the flu and…
I attended my final class at the University of Southern California (USC) last Wednesday, and I thought this would be a good time to look back at the ups and downs of navigating college with a disability. The support of those around me…
A signaling pathway determines whether muscle stem cells differentiate and mature into working muscle cells, an early study in mice found. Targeting this pathway and its two proteins — Fam3a and Stat3, which works to stimulate Fam3a — may treat diseases or illnesses of muscles, including muscular dystrophies. Getting these stem cells to…
Fulcrum Therapeutics announced it has acquired global rights to develop and potentially market losmapimod, an investigational treatment for facioscapulohumeral muscular dystrophy (FSHD), and that it plans to open a Phase 2 clinical trial testing the compound’s safety and efficacy this year. The compound was initially developed by GlaxoSmithKline (GSK) for…
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