Family Stories

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

Shanghai Jiao Tong University School of Medicine Begins Clinical Trials of LE051 Exon 51 Skipping Therapy

Research March 30, 2025 0

The purpose of LE051 study is to evaluate the...

Translarna (Ataluren) Rejected by EMA: Marketing Authorization Not Renewed

Press Releases March 29, 2025 0

In May 2024, PTC Therapeutics had been announced that...

DMD WarrioR’s Turkish Representative Shares His Views on Elevidys Gene Therapy: Is It Effective and Why Is It Expensive?

Editorials March 28, 2025 0

The efficacy of Elevidys in treating Duchenne muscular dystrophy...

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...

Cures of Duchenne (List of All Researches)

Research September 20, 2024 14

Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

DMD Warrior Mark Penzes from Hungary and His Family’s Determination to Receive Treatment

South Korean Trending News: Father Walks 740 km for Daughter with Duchenne Muscular Dystrophy

The Story of Aziz, DMD Warrior from Turkey

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