The U.S. Food and Drug Administration (FDA) has granted orphan drug status to elamipretide for treating Duchenne muscular dystrophy (DMD). According to Stealth BioTherapeutics, the therapy’s developer, elamipretide has been found to help improve the function of mitochondria — the powerhouses of cells — that…
Summer! The best time of year for my kids. We are currently counting down the days left of school. In Nebraska, school is out for summer break by the middle of May. So next week, all my children will be on summer break! My kids will spend plenty of…
The Spectrum Health Helen DeVos Children’s Hospital (HDVCH) in Michigan has been recognized as providing the highest quality of care for patients with Duchenne muscular dystrophy. Located in Grand Rapids, the hospital has become the first in the state to receive a certification from Parent Project Muscular Dystrophy…
An international team of scientists has developed a novel exon-skipping therapy mixture that has the potential to treat more than 40% of people with Duchenne muscular dystrophy (DMD). The therapy was described in a study, “Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the…
There are a lot of things to hate about Duchenne muscular dystrophy. Last weekend, I found myself uncontrollably crying while FaceTiming a friend as I was explaining why I wanted to be in a relationship before I lost the ability to move my muscles at all. It’s hard to…
I had one of those moments that hit you hard with a dose of the future last week. And those hits hurt. I was pushing my baby daughter, Callie, in her stroller. We were on a walk to the middle school my son Rowen attends. I happily thought about it…
To help support its popular summer camp and the neuromuscular disease community, the Muscular Dystrophy Association (MDA) has launched campaigns through October that involve more than 1,000 retailers nationwide. Proceeds from the MDA’s summer retail campaigns will benefit research, care, and advocacy for those living with muscular…
The U.S. Food and Drug Administration (FDA) has given Pfizer the go-ahead to open U.S. sites for the Phase 3 CIFFREO clinical trial, testing the experimental gene therapy PF-06939926 in boys with Duchenne muscular dystrophy (DMD). The FDA had placed a hold on Pfizer’s application to start the trial in…
I write my columns on Tuesdays. I do this because I procrastinate. My deadline is Wednesday morning, and I’m not about to wake up early on Wednesdays to get it done. I am not a morning person, but I’m too old and have too many kids to stay up late…
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…
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