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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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Duchenne Muscular Dystrophy (DMD) Exon 51 Skipping Therapies: A Promising Cures and Clinical Trials

Research 0
Duchenne Exon 51 skipping therapies are innovative treatments aimed...

Shanghai Jiao Tong University School of Medicine Begins Clinical Trials of LE051 Exon 51 Skipping Therapy

Research 0
The purpose of LE051 study is to evaluate the...

Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed

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In May 2024, PTC Therapeutics had been announced that...

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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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