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Pulmonology
Scientists have shown that src kinase inhibitors are effective at treating CF patients with a frame deletion of F508 in the CFTR gene.
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Metabolic disorder
Therapy for neuronopathic Gaucher Disease using gene therapy and a specialized neuronal lineage iPS cell that facilitates entry to and engraftment in the brain.
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"Oncology, Inflammation"
Pharmacologic and genetic inhibition of UBE2N suppresses the function and viability of MDS/AML cells lines and patient samples. A commercially available and a novel compound has been identified.
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Dermatology
Rapid reliable method for collection of keratinocyte RNA, DNA, microbiome, proteins, lipids, and metabolites.
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Oncology & Pulmonology
Increased FOXM1 is associated with proliferative diseases such as cancer and pulmonary diseases. A small molecule FOXM1 inhibitor has been identified.
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Pulmonology
A recombinant antibody has been developed for Cela1, which is important in diseases of stretch-induced distal airspace destruction such as emphysema.
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Surgery
Spheric endo-luminal traction device helps improve surgical outcomes for individuals with long-gap esophageal atresia
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Neurology
Dantrolene is an antagonist of ryanodine receptors (Ryrs) and blocks Ryr-mediated endoplasmic reticulum calcium release. It is an FDA-approved drug.
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Pharmacology
Cloud-based, HIPAA-compliant clinical decision-support platform that brings pharmacometrics and model-informed precision dosing directly to the bedside.
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Drug Delivery & Formulation
A validated algorithm to calculate when the next dose of an inhibitor of complement-mediated (C5) activity is required.