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I always try to pause to appreciate the small victories
I’m currently continuing to do the exercises my physical therapist recommended during my last therapy session. However, it hasn’t been stopping the progression of my facioscapulohumeral muscular dystrophy (FSHD). While I’m hoping the exercises are slowing the progression down, it seems like it gets harder every day for me to move. This relentless progression has…
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Supporting artists with disabilities through a new business venture
Three weeks ago, my girlfriend, Amanda, and I embarked on a unique journey by co-founding Rebirth Ensemble (RE), an art-making business with a distinct purpose. This partnership is deeply rooted in our shared passion for visual art, which has been a source of joy but also a progressively tricky challenge due to her visual impairment…
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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…
