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CureDuchenne Celebrates FDA’s Launch of Rare Disease Innovation Hub
We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new initiative represents a significant step forward in enhancing support and treatment options for rare disease patients, particularly those living with Duchenne and Becker muscular dystrophy.…
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DMD treatment SAT-3247 moves toward 1st trial
Satellos Bioscience is gearing up to launch a Phase 1 clinical trial of its Duchenne muscular dystrophy (DMD) treatment SAT-3247. The company said it has submitted an application to authorities in Australia seeking permission to start the trial, which is expected to test the safety and pharmacological properties of the oral treatment in healthy volunteers.…
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Support World Duchenne Day 2024 with CureDuchenne
As World Duchenne Day (WDD) approaches on September 7, 2024, we at CureDuchenne invite you to join us in raising awareness and showing support for our Duchenne community. This year, we have prepared a special social media graphics package for you. How to Support World Duchenne Day Post on Social Media Take a Photo Use…
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First oculopharyngeal MD patient sees better swallowing with BB-301
Treatment with the gene therapy BB-301 led to improved swallowing for the first person with oculopharyngeal muscular dystrophy (OPMD) to receive the treatment as part of an ongoing clinical trial, according to new interim data from BB-301’s developer Benitec Biopharma. “We are pleased to report continued positive interim clinical study data for Subject 1 in…
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My body clock ticks louder because of my Duchenne MD
Nearly two years ago, I authored a column for this website about my reflections on the semi-autobiographical rock musical “tick, tick … BOOM!” from American composer, lyricist, and playwright Jonathan Larson (best known for “Rent”). I’d just watched the 2021 film adaptation, directed by Larson’s fellow Broadway legend Lin-Manuel Miranda and distributed by Netflix. I…
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PPMD Applauds FDA Launch of Rare Disease Innovation Hub — A Milestone for Rare Disease Patients
Parent Project Muscular Dystrophy (PPMD) welcomes the recent announcement by the United States Food and Drug Administration (FDA) of the “Rare Disease Innovation Hub”. This initiative marks a pivotal moment in the journey towards better support and treatment for rare disease patients, including those living with Duchenne and Becker muscular dystrophy. In June 2024, PPMD…
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A Father Rises in Support of His Son
A Father Rises in Support of His Son: On this powerful episode of the “In Sickness” podcast, dad, Josh Argall opens up about the emotional rollercoaster following his son Devin’s diagnosis with Duchenne muscular dystrophy. From misdiagnoses to finding hope, Josh shares his journey and his determination to fight for his son’s future. Special guest…
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Losmapimod improved, stabilized FSHD upper limb function: Study
One year of losmapimod taken twice a day improved or stabilized upper limb function and muscle strength in adults with facioscapulohumeral muscular dystrophy (FSHD), according to an open-label pilot study. Blood and muscle tests also showed the therapy candidate successfully engaged with its intended target and had a favorable safety and tolerability profile. Details of…
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Fun summer activities that are still doable with Duchenne
I often read social media posts from moms asking for safe and fun ideas for their children to enjoy during summer break. When my boys were younger, I worried about that as well. I wanted my three sons with Duchenne muscular dystrophy (DMD) — Max, 18, Rowen, 15, and Charlie, 13 — to have the…
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NHL All-Star Ryan Getzlaf Returns to the Links for the 13th Annual Getzlaf Golf Shootout on September 13-14, Benefiting CureDuchenne
Event Has Raised More Than $5.8 Million Over the Last 12 Years to Drive a Cure for Duchenne Muscular Dystrophy NEWPORT BEACH, Calif., (July 12, 2024) — CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, will partner once again with NHL All-Star and retired Anaheim Ducks captain…
