DMD

Types of DMD Genetic Variants

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Duchenne Muscular Dystrophy (DMD) is a devastating genetic disorder primarily caused by various types of DMD genetic variants within the dystrophin gene, one of...

Genetic Causes of Duchenne Muscular Dystrophy

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Duchenne muscular dystrophy (DMD) is rooted in the intricate world of genetics, specifically emanating from changes in a single gene known as the Dystrophin...

What is Duchenne Muscular Dystrophy?

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Duchenne Muscular Dystrophy (DMD) is a genetic disorder that affects thousands of families worldwide. It primarily impacts boys, causing muscle degeneration and weakness over...

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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

UK Approves Givinostat (Duvyzat) for Duchenne Muscular Dystrophy Treatment

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Givinostat conditionally approved to treat patients with Duchenne muscular...

Santhera Announces Approval in Hong Kong for AGAMREE® (Vamorolone) as a Treatment for Duchenne Muscular Dystrophy

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Santhera Pharmaceuticals announces that the Department of Health of...

Researchers Discover 18-Digit Code (Wnt7a) that Allows Proteins to Hitch a Ride Around the Body

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Researchers from the University of Ottawa and The Ottawa...

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Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy

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

Capricor Therapeutics Submits Application for Treatment of DMD Cardiomyopathy

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Capricor Therapeutics, a biotechnology company developing transformative cell and...

Cure of Duchenne (Research Overview)

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Dystrophin Gene Therapy Mechanism Company Drug Status Micro-dystrophin Gene Replacement Sarepta Therapeutics Elevidys/SRP-9001 Approved Micro-dystrophin Gene Replacement Pfizer PF-06939926 Discontinued Micro-dystrophin Gene...

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