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Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne
Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and exon-skipping technology for individuals with Duchenne amenable to exon 51 skipping. According to Sarepta, this decision was informed by information…
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WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)
Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment of Duchenne. Satellos presented on the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem cells to repair and regenerate muscles. The session also includes a Q&A portion where Satellos addresses questions from the community…
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Phase 1/2 trial of del-brax enrolling new, possibly pivotal FSHD group
A new patient group is being enrolled in a Phase 1/2 trial of delpacibart braxlosiran (del-brax), an experimental and potentially disease-modifying therapy for facioscapulohumeral muscular dystrophy (FSHD), its developer, Avidity Biosciences, announced. The additional trial cohort, which is expected to be recruited in full early next year, aims to evaluate the effects of del-brax on…
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As a Duchenne mom, I know I can do hard things
The seasons are changing in central Nebraska. The leaves have turned yellow and orange, a beautiful contrast littered across the green lawns and familiar streets surrounding my home. The mornings are brisk, and I’ve finally pulled out my warmer sweaters and fuzzy socks. I’ve been drinking pumpkin spice coffee for weeks! However, the weather here in…
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CureDuchenne partners on World of Warcraft research fundraiser
CureDuchenne is partnering with Blizzard Entertainment, maker of the massively multiplayer online role-playing game World of Warcraft, on a fundraiser to help advance research into new treatments for Duchenne muscular dystrophy (DMD). Through Jan. 7, players of the video game will have the opportunity to adopt an in-game pet fox named Reven, who will accompany…
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My trip to the Idlewild amusement park was wild, but worth it
Idlewild, which is near Ligonier, Pennsylvania, is an old-style amusement park that’s been around since the days when the gentry from Pittsburgh took the train to their summer homes in the foothills of the Laurel Highlands. It’s a wonderful park for all ages, but it’s absolutely perfect for younger children like my grandkids, Iva Jane,…
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FDA Accepts Translarna NDA Resubmission for Review
A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the FDA will now carefully evaluate the data to determine ataluren’s potential for approval. According to PTC, the NDA resubmission is…
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Flu Season Ready: Protecting Yourself and Your Family
With the start of school and the onset of winter, we can expect a rise in respiratory viruses. This year, it’s crucial to safeguard yourself, your children, and your family from all respiratory infections, particularly the more severe ones like influenza (flu), RSV, and COVID. Because respiratory muscles can be weakened in individuals with Duchenne…
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FDA awards LAMA2-CMD therapy MDL-101 orphan drug status
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The designation is intended to encourage the development of therapies for serious or life-threatening rare diseases, which are those affecting fewer than 200,000 people in the U.S. The designation…
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Duchenne affects my entire life, down to a DIY project
I’m a mom to seven children: Lexi, 23; Max, 18; Chance, 17; Rowen, 15; Charlie, 13; Mary, 10; and Callie, 2. As part of that job, I’m also a caregiver to Max, Rowen, and Charlie, who all have Duchenne muscular dystrophy (DMD). Additionally, I’m a chronic DIYer (do-it-yourselfer). Yes, you read that correctly. Perhaps you’re…
