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Holtz Children’s Hospital

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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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Dr. Aravindhan Veerapandiyan, a neurologist at Arkansas Children’s Hospital, Reviews RGX-202 Gene Therapy Study

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At the American Academy of Neurology Annual Meeting, which...

Givinostat EMA Approval: When Will Givinostat (Duvyzat) Be Approved in Europe?

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Givinostat has already received regulatory approval in the US...

Satellos SAT-3247 Stem Cell Approach May Restore Muscle Regeneration in Duchenne Muscular Dystrophy

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Genetic interventions such as exon skipping or gene therapy...

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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)...

What is Duvyzat (Givinostat)?

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

In Which Countries Is Elevidys Approved? Is Elevidys Approved In Europe?

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Elevidys is the only approved gene therapy in the...

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