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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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Metagenomi’s New CRISPR System (VK CAST) Enables Large Gene Integration

Research 0
Metagenomi, a precision genetic medicines company committed to developing...

Why Isn’t Roche Applying Marketing Authorization in Every Country for Elevidys Gene Therapy?

Editorials 0
Gene therapies, particularly those like Elevidys, have the potential...

Hydrogel Delivery System Holds Potential for Cell-Based Therapy of Duchenne Muscular Dystrophy

Research 0
In a mouse model of muscular dystrophy, researchers in...

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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)...

Cures of Duchenne (List of All Researches)

Research 12
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 0
Capricor Therapeutics, a biotechnology company developing transformative cell and...

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