Dystrophin Gene Mutation News, Researches, Clinical Trials and Articles

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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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Mutations Amenable to Exon 50 Skipping Therapies in Duchenne Muscular Dystrophy

DMD July 28, 2026 0

Mutations amenable to exon 50 skipping are an important...

GEN6050X Exon 50 Skipping Base Editing Drug: One-Year Results Show Encouraging Progress for Duchenne Muscular Dystrophy

Press Releases July 25, 2026 0

The GEN6050X exon 50 skipping base editing drug, developed...

Sarcomatrix Plans First Human Trial of S-969 for Duchenne Muscular Dystrophy in 2027

Research July 24, 2026 0

For families affected by Duchenne muscular dystrophy (DMD), every...

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Therapies December 26, 2024 0

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

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Editorials April 10, 2026 0

The landscape of potential new gene therapies for Duchenne...

The Brutal Truth: Why Duchenne Muscular Dystrophy Treatment Costs Are Out of Control

Editorials April 6, 2026 0

The duchenne muscular dystrophy treatment cost has become one...

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