HomeTagsGolodirsen

Golodirsen

Editorials

Vyondys 53: What Does FDA Rejection Letter Mean?

Popular

Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

DMD Warrior - December 26, 2024

Must Read

Press Releases

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

Press Releases

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

Press Releases

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

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.

Latest Articles

INS1201 Gene Therapy Phase 1 Clinical Trial Begins

Research July 17, 2025 0

Insmed was announced on the page published on ClinicalTrials.gov...

Sarepta to Lay Off About 500 Employees After Duchenne Gene Therapy Setbacks

Press Releases July 17, 2025 0

Sarepta stated that company intends to halt development on...

Precision BioSciences Highlights New Preclinical Data for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

Press Releases July 16, 2025 0

Precision BioSciences a clinical stage gene editing company utilizing...

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 5

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

Duchenne Muscular Dystrophy: Treatment & Cost

Editorials January 2, 2025 1

The excitement you feel until your child is born...

Golodirsen

Must Read

About us

Latest Articles

Most Popular