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A Safety Update on ELEVIDYS – June 2025
We are heartbroken to learn of the loss of another member of our community following […]
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Sarepta pauses Duchenne gene therapy for non-ambulatory individuals after second death due to acute liver failure
CureDuchenne is sad to share that another person has passed away due to acute liver […]
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MDA Ambassador Guest Blog: How Making an Impact Renewed My Purpose After FSHD Diagnosis
Ranae Beeker, who is in her late 60s, has found her greatest joy in her […]
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REGENXBIO WEBINAR
AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data Watch HERE The post REGENXBIO WEBINAR appeared […]
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Our family’s all-star football player also lives with Duchenne
I’m a mother of seven and a caregiver to the three of my sons who […]
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PPMD Announces Children’s Hospital of Philadelphia as CDCC, Recipient of Inaugural Clinical Research Designation
PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) […]
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Duvyzat conditionally approved by EC to treat Duchenne MD
The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating […]
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Capricor’s Advisory Committee meeting with FDA to be held July 30, 2025
Capricor Therapeutics, which received early funding from CureDuchenne, announced regulatory updates for their Deramiocel program […]
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After incredible hardships, I still feel blessed
I’m between two anniversaries. Today is the 36th anniversary of the collision I had with […]
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Viral vector in LGMD2E gene therapy gets new FDA designation
The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector […]
