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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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Nippon Shinyaku Releases First Clinical Trial (First in Human) of Brogidirsen NS-089/NCNP-02 for the Treatment of Duchenne

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The National Center of Neurology and Psychiatry is announced...

What is Ataluren (Translarna)? What is Ataluren Used For?

Research 0
Ataluren (Translarna) is an investigational drug used to delay...

Avidity Biosciences Prepares for Three Rare Muscle Disease Programs’ 2025 BLA Submission and Commercialization

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A biopharmaceutical firm dedicated to developing a novel class...

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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)...

Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy

Research 0
Capricor Therapeutics, a biotechnology company developing transformative cell and...

Cures of Duchenne (List of All Researches)

Research 2
Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

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