Exondys 51 News, Researches 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.

Latest Articles

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...

Exondys 51

Must Read

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

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

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