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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

Three Ongoing Clinical Trials of Elevidys Gene Therapy Temporarily Halted in Europe

Press Releases April 1, 2025 0

Three ongoing clinical trials of ELEVIDYS (delandistrogene moxeparvovec-rokl), a...

Keros Therapeutics Announces Initial Topline Results from the Phase 1 Clinical Trial of KER-065 in Healthy Volunteers

Research March 31, 2025 0

Keros Therapeutics, a clinical-stage biopharmaceutical company focused on developing...

Duchenne Muscular Dystrophy (DMD) Exon 51 Skipping Therapies: A Promising Cures and Clinical Trials

Research March 31, 2025 0

Duchenne Exon 51 skipping therapies are innovative treatments aimed...

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...

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Must Read

Frequently Asked Questions About Solid Biosciences SGT-003 Gene Therapy

Stitch RNA: New Method for Duchenne Gene Therapies

Solid Biosciences Plans to Request a Meeting with the FDA in mid-2025 to Discuss Potential Accelerated Approval Pathways for SGT-003