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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.

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Regenxbio Reports Positive Biomarker Data From Affinity Duchenne Trial Of Rgx-202 Gene Therapy

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REGENXBIO reported new, positive interim data from two additional...

Satellos Presents Initial Data from the Phase 1 Trial of SAT-3247 at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference

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Satellos Bioscience, a biotech company developing new small molecule...

What is Cytomegalovirus Infection (CMV)?

DMD 0
Sarepta shares that a teenager with Duchenne muscular dystrophy...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

Approved Treatments 5
A genetic condition known as Duchenne muscular dystrophy (DMD)...

Cures of Duchenne (List of All Researches)

Research 13
Dystrophin Gene Therapy MechanismCompanyDrugStatus Micro-dystrophin Gene Replacement Sarepta TherapeuticsElevidys/SRP-9001ApprovedMicro-dystrophin Gene ReplacementPfizerPF-06939926DiscontinuedMicro-dystrophin Gene...

What is Duvyzat (Givinostat)?

Approved Treatments 1
For individuals with Duchenne muscular dystrophy (DMD), adults and...

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