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Muscular Dystrophy Association

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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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After Second Patient Death, Duchenne Muscular Dystrophy Communities Want Answers About Elevidys Gene Therapy

Editorials 0
In a report released May 14 in Neurology, the...

Capricor Therapeutics Announces BMD Orphan Drug Designation and DMD Program Regulatory Development for Deramiocel (CAP-1002)

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

Sarepta Reported Elevidys Gene Therapy for Duchenne Muscular Dystrophy will not be used in non-ambulatory patients

ELEVIDYS Press Releases 0
ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for...

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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 5
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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