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Exon Deletions

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What is Exon Deletion in Duchenne Muscular Dystrophy (DMD)? What Happens as a Result of Exon Deletion?

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Types of DMD Genetic Variants

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD Warrior - December 26, 2024

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Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne Muscular Dystrophy

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Shanghai Jiao Tong University School of Medicine Begins Clinical Trials of LE051 Exon 51 Skipping Therapy

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Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed

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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 June 8, 2025 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 June 6, 2025 0

The Regenxbio press statement stated that the findings, which...

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

Press Releases May 29, 2025 0

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

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 3

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...

Exon Deletions

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