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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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Scribe Therapeutics and Prevail Therapeutics to Share In Vivo CRISPR-based Genome Editing Data at ASGCT 2025

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Scribe Therapeutics, a genetic medicines company unlocking the potential...

Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy

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Capricor Therapeutics , a biotechnology company developing transformative cell...

The Dystrophin Gene: Function, Disorders, and Therapeutic Advances

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The dystrophin gene is a vital component of human...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments 5
A genetic condition known as Duchenne muscular dystrophy (DMD)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research 3
The results of new gene therapy studies for Duchenne...

What is Duvyzat (Givinostat)?

Approved Treatments 1
For individuals with Duchenne muscular dystrophy (DMD), adults and...

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