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Caregiving for sons with DMD is like being a frog in boiling water
One of my favorite early-summer memories is from when my sons Max and Rowen were younger. Both lovely little boys — one blond-haired and blue-eyed, the other dark-haired and brown-eyed — squealed with laughter upon discovering a frog in our front yard. Max held the frog up in his hands and away from our yellow…
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Next-gen gene therapy SGT-003 named FDA orphan drug
SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined as conditions affecting fewer than 200,000 people…
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Astellas Releases Results of Discontinued Phase 1b Study of ASP0367
Astellas has shared results of the Phase 1b study of ASP0367 (MA-0211), an oral investigational drug. The study aimed to evaluate safety, tolerability, and preliminary efficacy in individuals living with Duchenne aged 8 to 16 years old. The study was stopped early, in November 2022, because not enough patients were available to take part in the study. While…
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PPMD Hosts 2024 Duchenne Healthcare Professionals Summit
Each January, PPMD brings together experts in the field for our Duchenne Healthcare Professionals Summit to ensure that caring for individuals with the latest information and technology remains a constant priority. Healthcare professionals from PPMD’s Certified Duchenne Care Centers and other neuromuscular programs providing dystrophinopathy care, as well as researchers, academics, and industry partners, gather…
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Turning Challenges into Opportunities: A Conversation With Justin Skeesuck From “I’ll Push You”
Justin Skeesuck, a 48-year-old Idahoan featured in the documentary “I’ll Push You,” started losing the use of his hands in 2010. So, in 2012, he moved to Italy. For a lot of people that might not make a lot of sense, especially given his situation. In 2002, Skeesuck was diagnosed with an autoimmune and neurological…
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