Celebrate? Yes, we do. There was a time when I didn’t think celebrating and Duchenne muscular dystrophy could coexist. I thought a life touched by Duchenne would be voided of happiness, joy, and celebration. I was very wrong. I have since learned that life with Duchenne just makes the…
The first healthy volunteer has been dosed with an investigational exon-skipping therapy, called PGN-EDO51, for Duchenne muscular dystrophy (DMD) in a Phase 1 trial, the treatment’s developer, PepGen, has announced. The single-ascending dose trial is investigating the treatment’s safety and tolerability in about 40 healthy adult males in Canada.
Santhera Pharmaceuticals and ReveraGen BioPharma will seek approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD) in the U.S. this year, with a filing expected to be completed by the end of June. The submission of the companies’ rolling new drug application (NDA) to the U.S. Food and Drug Administration…
During Mass on Sunday, the priest talked about an aspect of faith that acts as an infusion for the soul. As a mother of three sons with Duchenne muscular dystrophy (DMD), I loved the analogy and was grateful because I thought it was something my sons would understand. All three…
Thirteen new hospitals and health institutions have been added to the Muscular Dystrophy Association’s MDA Care Center Network, expanding the network’s reach across the United States. These additions, which comprise more than 150 care centers, include 10 new grant awards as well as three care affiliates that have been…
Electrical muscle stimulation that mimics resistance training exercises improves muscle structure, function, and survival rates in a Duchenne muscular dystrophy (DMD) zebrafish model, a new study reveals. “The longstanding consensus in the muscular dystrophy field is that minimizing resistance training preserves muscle strength and mass because it lowers the…
I’ve had a conversation with my son Rowen, 13, that we continue to revisit, and I feel like it’s something all parents raising kids with Duchenne muscular dystrophy deal with on some level. How do we teach our children to advocate for themselves? My three sons with Duchenne all…
When it comes to making decisions about how care goals should change for adolescent and young adults with Duchenne muscular dystrophy (DMD) as the disease progresses, informal conversations among family members are more common than discussions with clinicians, according to a study based on interviews with DMD patients and…
An ion channel called Piezo1 regulates muscle stem cell (MuSC) states and the number of cells that respond quickly to muscle injury, promoting muscle repair, a study in mice shows. Notably, this ion channel was found to be poorly activated in a mouse model of Duchenne muscular dystrophy (DMD), and…
When we think about fighting, we might imagine the Ultimate Fighting Championship ring, where men and women beat each other up in front of millions of fans. Or we might see soldiers firing machine guns, or Darth Vader and Luke Skywalker trading blows with lightsabers. Our minds probably…
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