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Approved Treatments

NICE Final Guidance for AGAMREE (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy Is Positive for Santhera

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD Warrior - December 26, 2024

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

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DMD WarrioR’s Turkish Representative Shares His Views on Elevidys Gene Therapy: Is It Effective and Why Is It Expensive?

Research

Fn14 Protein May Protect Muscle Stem Cells, Offering Promise for Development of Duchenne Treatments

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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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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...

Entrada Therapeutics Receives EU Authorization to Start Exon 45 Skipping Clinical Trial in Duchenne Muscular Dystrophy

Press Releases May 28, 2025 0

Entrada Therapeutics, today announced it has received authorization from...

Entos Pharmaceuticals Develops Re-Dosable, Full-Length Dystrophin Gene Therapy: Fusogenix PLV Method

Press Releases May 22, 2025 0

Entos Pharmaceuticals Inc. (Entos) is a biotech business that...

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...

What is Duvyzat (Givinostat)?

Approved Treatments October 13, 2024 1

For individuals with Duchenne muscular dystrophy (DMD), adults and...

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