Masters now has an increasing supply of Deflazacort (a steroid available to treat Duchenne outside the US). However, due to recent regulatory changes, families should now allow 3…

Grant Will Be Used to Support Pilot Study for X-Ar Exoskeletal Arm Technology

Parent Project Muscular Dystrophy has awarded Talem Technologies a grant for over $70,000 to support a pilot study of their…

Presentations from Day 2 of PPMD’s 21st Annual Connect Conference in Washington, D.C. are now available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next couple of weeks. To start, check out the presentations from Day 2…

The first batch of presentations from PPMD’s 21st Annual Connect Conference in Washington, D.C. are available for download. If you were unable to attend this year’s conference or if you want to replay a presentation that you found particularly useful, we will be sharing all of the presentations over the next couple of weeks. To start, check…

For over a year, PPMD has been working with Congress on landmark legislation called 21st Century Cures. The bill aims to speed the discovery, development, and delivery of therapies for diseases that have an unmet medical need like Duchenne. We have been thankful to Congress for their willingness to meaningfully engage the patient…

Historically, people with Duchenne have been told that using inhaled anesthetics can cause malignant hyperthermia (“MH”; extremely high body temperature), a condition causing rhabdomyolysis (breakdown of skeletal muscle tissue causing the release of myoglobin, a muscle protein that damages the kidneys) and…