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Capricor will file this year for FDA approval of Deramiocel for the treatment of Duchenne cardiomyopathy
As an early investor in Capricor Therapeutics, CureDuchenne is happy to share that Capricor has […]
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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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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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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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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 […]
