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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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High Anti-AAV Antibodies May No Longer Be a Barrier to Elevidys Administration

Press Releases 0
One of the most significant obstacles to the application...

MetrioPharm’s MP1032 Receives EMA Orphan Drug Designation in Duchenne Muscular Dystrophy

Press Releases 0
MetrioPharm announced today that the European Medicines Agency has...

Genethon to Launch Pivotal Trial in Europe of GNT0004 a Low-Dose Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy

Research 1
Genethon has received approvals from regulatory authorities, MHRA and...

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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)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research 6
The results of new gene therapy studies for Duchenne...

Duchenne Muscular Dystrophy: Treatment & Cost

Editorials 1
The excitement you feel until your child is born...

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