European Medicines Agency

About us

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.

Latest Articles

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

DMD February 5, 2025 0

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

Research February 5, 2025 0

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

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

Research February 4, 2025 0

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

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD December 26, 2024 1

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

Cures of Duchenne (List of All Researches)

Research September 20, 2024 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

Research October 10, 2024 0

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

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