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Mutations Amenable to Exon 50 Skipping Therapies in Duchenne Muscular Dystrophy

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Duchenne muscular dystrophy (DMD) is a severe genetic disorder...

BBM-D101 Gene Therapy Phase 1/2 Clinical Trial Begins

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Belief BioMed (BBM), headquartered in China, is a global...

JCR Pharmaceuticals Proprietary JUST-AAV Capsids May be Used in the Development of Duchenne Cures

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Alexion, AstraZeneca’s rare disease subsidiary, is licensing specialized gene...

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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 5
The results of new gene therapy studies for Duchenne...

Duchenne Muscular Dystrophy: Treatment & Cost

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The excitement you feel until your child is born...

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