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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

The Dystrophin Gene: Function, Disorders, and Therapeutic Advances

DMD May 1, 2025 0

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

Tevard Biosciences to Present Data Demonstrating tRNA-based Therapy Rescued Full-Length Dystrophin and Motor Function in Duchenne Muscular Dystrophy Model

Research April 30, 2025 2

Preclinical results demonstrating the potential of Tevard Biosciences' tRNA...

EMA Recommends EU Approval for Duvyzat (givinostat) to Treat Duchenne Muscular Dystrophy

Approved Treatments April 25, 2025 0

The European Medicines Agency (EMA) has suggested that Duvyzat...

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

Precision BioSciences Presents Preclinical Efficacy and Durability Data on PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) at the 2025 Muscular Dystrophy Association...

Sarepta, Reports 16 Years Old Boy Death After Elevidys Gene Therapy Treatment

Givinostat’s Exclusive Distribution Agreement in 17 Central and Eastern European Countries, Including the Baltic States, has been Announced by Italfarmaco and Medis