Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the University of Missouri to help better understand and improve the treatment of cardiac disease in people with Duchenne muscular dystrophy (DMD). The funding falls under PPMD’s…
I have spent my entire career working and volunteering in the nonprofit arena. My focus has moved from mental health to aging to disabilities. I learned from each step and took that knowledge into the next phase of my life. There were always things I could use in the future.
Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (DMD), real-world data show. The most recent results from the ongoing STRIDE registry — an observational study across more than 50 care centers in Europe and Israel — were presented at…
Sometimes life with a chronic invisible illness can feel backward and upside down. As if anything that I do, whether positive or negative, could come back to bite me in the butt. It’s dizzying to try to predict people’s reactions to something as simple as a photo of me showing…
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (DMD). PIM designation indicates that the MHRA considers vamorolone likely to offer major therapeutic advantages for DMD patients. The MHRA will next…
A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne muscular dystrophy (DMD) without triggering an immune response, a new study reports. The study, “Non-immunogenic…
Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD) being developed by ReveraGen Biopharma, continues to improve muscle function in DMD patients with fewer side effects than standard corticosteroids, topline data in 23 boys in an ongoing Phase 2 trial show. These findings were detailed in a late-breaking…
“That’s right. You’ll be able to resume your regular diet after gallbladder removal.” My surgeon’s words ring in my ears as my television airs food commercials and I chomp on carrot sticks. Two weeks after my operation and I’m finally back to my old self. I can flaunt my skinny…
I recently participated in a panel discussion at CureDuchenne 2019 Futures titled “Friends.” I gave a brief presentation, along with other members of the disease community, on the importance of friendship. From the responses we received, it was clear to us that many parents present at the event were…
To address the need in precision medicine for human biological specimens, CureDuchenne is creating a biobank to provide Duchenne muscular dystrophy (DMD) researchers with a collection of blood and skin tissue samples. The overarching goal is to use the biorepository to fuel and advance DMD research. The CureDuchenne Biobank…
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