Exon Mutations 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

SonoThera Ultrasound-Mediated Delivery Method Can Deliver Full-Length Dystrophin to Cells

Press Releases May 7, 2025 0

The findings of the study, which are currently being...

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

Press Releases May 5, 2025 0

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

Press Releases May 5, 2025 0

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

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...

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Must Read

Regenxbio Reports Positive Biomarker Data From Affinity Duchenne Trial Of Rgx-202 Gene Therapy

Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

What is Cytomegalovirus Infection (CMV)?