GNT0004 Gene Therapy 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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Latest Articles

Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen, an Exon 51 Skipping Therapy for Duchenne Muscular Dystrophy (DMD)

Press Releases July 20, 2026 0

Families affected by Duchenne muscular dystrophy (DMD) received encouraging...

Santhera Expands Biomedica Agreement to Extend AGAMREE (Vamorolone) Distribution Across Central Asia and the Caucasus

Press Releases July 13, 2026 0

Santhera today announces an expansion of its exclusive distribution...

Creatine Kinase Level in Duchenne Muscular Dystrophy: Normal Ranges, High CK Levels, Diagnosis, Blood Tests & FAQs

DMD July 10, 2026 0

A creatine kinase level in Duchenne muscular dystrophy (DMD)...

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Therapies December 26, 2024 4

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

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Editorials April 10, 2026 9

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 2

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

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Why CK, AST, and ALT Data Matter More Than Videos in Duchenne Gene Therapy

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ELEVIDYS ENHANCE Study: A Phase 4 Trial Begins to Evaluate Safety and Effectiveness in Duchenne Muscular Dystrophy