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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.

Latest Articles

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

Editorials June 19, 2025 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)

Press Releases June 17, 2025 0

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 June 15, 2025 0

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

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments December 26, 2024 5

A genetic condition known as Duchenne muscular dystrophy (DMD)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research January 5, 2025 5

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

Duchenne Muscular Dystrophy: Treatment & Cost

Editorials January 2, 2025 1

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

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Must Read

U.S. Department of Defense Announces Duchenne Muscular Dystrophy Research Programs and Funding Opportunities for Fiscal Year 2025

Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe

Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers