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Method for treating glycogen storage disease

The present disclosure is directed to methods of treating a...


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

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


Muscle mass influences upon response to therapy

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


Gene therapy for ATP1A3-Related Neurologic Disorders

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


A tool to facilitate high affinity binding between beta-Arrestin and GPCR

Unmet Need Beta-arrestins are a highly conserved family of cytosolic...


Small molecule therapeutics for Kabuki syndrome and related disorders

Unmet Need Kabuki syndrome is a developmental disorder characterized by...


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...