PBGENE-DMD Therapy News, Researches, Clinical Trials and Articles

About us

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.

Powered by Hostinger

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...

Duchenne MD

Genetic

Care Guidelines

Health Guidelines

PBGENE-DMD Therapy

Must Read

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

Does My Child Have Duchenne Muscular Dystrophy (DMD)?

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