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Method for treating glycogen storage disease
The present disclosure is directed to methods of treating a...
MORE >MEK inhibitors as therapeutic agents and diagnostic tools for hemoglobinopathies, including thalassemia
Value Proposition Patients with sickle cell disease experience acute vaso-occlusive...
MORE >A method of screening and monitoring glycogen storage diseases with a single biomarker
Unmet Need Glycogen storage disease type I (GSD I) is...
MORE >Muscle mass influences upon response to therapy
Duke University is seeking a company interested in commercializing a...
MORE >Gene therapy for ATP1A3-Related Neurologic Disorders
Unmet Need Children with Alternating Hemiplegia of Childhood can have...
MORE >A tool to facilitate high affinity binding between beta-Arrestin and GPCR
Unmet Need Beta-arrestins are a highly conserved family of cytosolic...
MORE >Small molecule therapeutics for Kabuki syndrome and related disorders
Unmet Need Kabuki syndrome is a developmental disorder characterized by...
MORE >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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