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We do not want children to die from a disease that can be cured. We want to give hope to families and ensure that all children with DMD are treated and regain their health.

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Call from Families to European Medicines Agency (EMA): Re-examine Ataluren (Translarna)

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Three parents representing 800 families and their children affected...

Russian Biotech Company Generium has Begun Clinical Trials of a Gene Therapy for Duchenne Muscular Dystrophy

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Clinical studies for a therapy for Duchenne muscular dystrophy...

Cumberland Pharmaceuticals Announces Fantastic Results from Phase 2 FIGHT Duchenne Trial in DMD Heart Disease

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Cumberland Pharmaceuticals, a specialty pharmaceutical company with development efforts...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD 1
A genetic condition known as Duchenne muscular dystrophy (DMD)...

Cures of Duchenne (List of All Researches)

Research 7
Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy

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Capricor Therapeutics, a biotechnology company developing transformative cell and...

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