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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

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...

Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

Press Releases April 24, 2025 0

Today, Dyne Therapeutics, a clinical-stage company dedicated to developing...

Agamree (vamorolone) will be Covered in Slovenia by the Slovenian Health Insurance Fund

Press Releases April 24, 2025 0

The Slovenian Health Insurance Institute (ZZZS) today added Agamree...

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)...

What is Duvyzat (Givinostat)?

Approved Treatments October 13, 2024 1

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

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research January 5, 2025 2

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

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Must Read

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

Biophytis’ BIO101 Therapy for Treatment of Sarcopenia Could Bring Hope to Duchenne Muscular Dystrophy Patients

Eteplirsen (Exondys 51) Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy