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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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What’s Next for Patients with Duchenne Muscular Dystrophy (DMD) After Pharma Giants Pull Back from AAV-Based Gene Therapy Research?

Editorials 0
Adeno-associated virus (AAV) vectors were once heralded as the...

Micro Dystrophin: 5 Critical Questions Everyone Is Thinking About

DMD 0
Micro dystrophin and full-length dystrophin play crucial roles in...

SonoThera Ultrasound-Mediated Delivery Method Can Deliver Full-Length Dystrophin to Cells

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The findings of the study, which are currently being...

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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)...

Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy

Research 3
The results of new gene therapy studies for Duchenne...

What is Duvyzat (Givinostat)?

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

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