Gene therapy for ATP1A3-Related Neurologic Disorders

Unmet Need Children with Alternating Hemiplegia of Childhood can have...

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Enhancing of homing and/or engraftment of cord blood mononuclear cells in the central nervous system

Value Proposition Leukodystrophies are inherited disorders resulting in decreased motor...

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MEK inhibitors as therapeutic agents and diagnostic tools for hemoglobinopathies, including thalassemia

Value Proposition Patients with sickle cell disease experience acute vaso-occlusive...

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A method of screening and monitoring glycogen storage diseases with a single biomarker

Unmet Need Glycogen storage disease type I (GSD I) is...

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Muscle mass influences upon response to therapy

Duke University is seeking a company interested in commercializing a...

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