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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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Ullrich Awareness Day
Finally, after years of battling and campainging, we now have an official Ullrich Muscular Dystrophy […]
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Protected: FDA Approval of ELEVIDYS: Key Takeaways from the CureDuchenne Webinar
This content is password protected. To view it please enter your password below: Password: The […]
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What I want people to know about FSHD and its progression
As I was finishing up a recent interview with the host of “FSHD Radio: Straight […]
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Twins offer insights into DM1 cognitive deficit contributors
A pair of identical twins with myotonic dystrophy type 1 (DM1) were found to have different […]
