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Orphan Drug

GEn1E Lifesciences and GEn-1123: A New Precision Medicine Approach for Duchenne Muscular Dystrophy (DMD)

European Commission Grants Orphan Drug Designation to SGT-003 for Duchenne Muscular Dystrophy

FDA Grants Orphan Drug Designation to NS-051/NCNP-04 for the Treatment of 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

Keros Announces U.S. FDA Orphan Drug Designation Granted to KER-065 for the Treatment of Duchenne Muscular Dystrophy

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

The FDA Designates PBGENE-DMD as an Orphan Drug by Precision BioSciences to Treat Duchenne Muscular Dystrophy

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

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Frequently Asked Questions About Elevidys Used for DMD

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

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Care Guidelines

Breathing Exercises in Duchenne Muscular Dystrophy: Respiratory Therapy, Pulmonary Care & Lung Support

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Latest Articles

NewBiologix and Synastra Partner to Advance DMD Gene Therapy Manufacturing

Press Releases September 2, 2026 0

A new international collaboration is bringing together biotechnology expertise...

What Is Exon Deletion? Understanding Exon Deletions in Duchenne Muscular Dystrophy

DMD September 2, 2026 0

What is exon deletion, and why does it matter...

tRNA Research for Nonsense Mutations Offers New Hope for Genetic Disorders

Research August 30, 2026 0

Researchers at the University of Toronto, University Health Network...

Most Popular

Frequently Asked Questions About Elevidys Used for DMD

Approved Therapies August 21, 2026 0

Duchenne muscular dystrophy (DMD) is a genetic condition that...

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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