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Approved Therapies

Approved Treatments for Duchenne Muscular Dystrophy

SQY Therapeutics Receives Orphan Drug Designation from the European Medicines Agency for Exon 51 Skipping Therapy SQY51 for Treatment of Duchenne Muscular Dystrophy

MetrioPharm’s MP1032 Receives EMA Orphan Drug Designation in Duchenne Muscular Dystrophy

The European Medicines Agency (EMA) Issued a Negative Opinion on Elevidys

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

Approved Therapies

EMA Recommends EU Approval for Duvyzat (givinostat) to Treat Duchenne Muscular Dystrophy

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

Givinostat EMA Approval: Is Givinostat (Duvyzat) Approved by EMA?

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

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Frequently Asked Questions About Elevidys Used for DMD

Can Ekinci - August 21, 2026

Must Read

Del-Zota SAFARI44 Phase 3 Trial: A New Exon 44 Skipping Study for Duchenne Muscular Dystrophy

RGX-202 Duchenne Gene Therapy Shows NSAA Improvement and Microdystrophin Expression — But Important Biomarker Data Missing

CRISPR GuideX Tool: hfCas12Max in CRISPR-Based Therapeutics

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Latest Articles

Beyond Micro-Dystrophin: Could Full-Length Dystrophin Be the Next Step in DMD Gene Therapy?

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Frequently Asked Questions About Elevidys Used for DMD

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