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Capricor Therapeutics Plans to File Biologics License Application for Full Approval of Deramiocel for the Treatment of Duchenne Cardiomyopathy
Capricor Therapeutics announced today its intent to file a Biologics License Application (BLA) seeking full […]
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How I fully embrace life with limb-girdle muscular dystrophy
I believe that all of us who live with a chronic illness deal with a […]
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Going gray prompts questions about the impact of caregiving on my life
I’m going gray. Wait, I should clarify that I’ve been coloring my hair since I […]
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DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease […]
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PPMD Contributes to National Academies Report on Rare Disease Regulatory Processes
We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus […]
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Setting Up A Successful School Year
Setting Up A Successful School Year PPMD is here to make sure that families within […]
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PPMD Community Webinar Recap: Overview of DUVYZAT and Access Resources
On September 18, 2024, PPMD hosted an informative community webinar featuring representatives from ITF Therapeutics, […]
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Overcoming mental hurdles as I transition to using a walker
I’ve had a walker with wheels for a decade. I got it after back surgery […]
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PPMD & Global Patient Advocacy Groups Award BIND Project $113,000 to Further Brain Involvement Research
PPMD is thrilled to announce that we have collaborated with a number of patient organizations […]
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Why patient advocacy is important for those of us with Duchenne
Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate […]
