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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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Miraculous Steroid Vamorolone (Agamree) Could Offer Safer Treatment for DMD

Research 0
A novel steroid medication that is used to treat...

U.S. Department of Defense Announces Duchenne Muscular Dystrophy Research Programs and Funding Opportunities for Fiscal Year 2025

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We extend our deepest gratitude to the Members of...

Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe

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Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments 5
A genetic condition known as Duchenne muscular dystrophy (DMD)...

What is Duvyzat (Givinostat)?

Approved Treatments 1
For individuals with Duchenne muscular dystrophy (DMD), adults and...

Cures of Duchenne (List of All Researches)

Research 14
Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

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