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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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European Commission Approves Duvyzat for the Treatment of Duchenne Muscular Dystrophy

Approved Treatments 0
The European Commission (EC) has given conditional marketing authorization...

New Positive Functional Data From The RGX-202 Affinity Duchenne® Trial Phase 1/2 Reported By Regenxbio

Regenxbio RGX-202 Press Releases 0
The Regenxbio press statement stated that the findings, which...

PepGen Announces It Will Halt Clinical Trials of Exon 51 Skipping Study

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PepGen, a clinical-stage biotechnology company advancing the next generation...

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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 3
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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