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Breakthrough in skeletal muscle regeneration
In a finding that opens the door to the development of targeted therapies for various muscle disorders, newly published research identifies key mechanisms of skeletal muscle regeneration and growth of muscles following resistance exercise.
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How a son’s MRI stirred up fields of dreams and hopes
This week, one of my boys had to get an MRI. Three of my four sons — Max, 18, Rowen, 15, and Charlie, 13 — are living with Duchenne muscular dystrophy (DMD), and they’ve had annual MRIs for the past 14 years (well, fewer for Charlie). Sometimes they’ve had more than one a year because…
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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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New gene therapy for muscular dystrophy offers hope
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles. The research focuses on delivering a series of protein packets inside shuttle vectors to replace the defective DMD…
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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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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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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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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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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…
