Award Data

DESCRIPTION provided by applicant The approval of the cystic fibrosis CF drug invocator for patients with the G D mutation validates CFTR modulators for improving clinical outcomes in CF For most CF patients with F del and other mutations however multi drug combination therapy will likely be necessary and sensitive biomarkers of augmented CFTR function in response to various agents ...

Abstract This Phase I STTR application proposes development of an innovative low cost magnetic levitation motor specifically designed for pediatric extracorporeal cardiac and cardiopulmonary therapies Magnetic levitation enables contact free impeller operation thereby eliminating critical areas of wear and heat generation that can contribute to hemolysis and thrombosis The extracorporeal pedia ...

DESCRIPTION provided by applicant Neurodegenerative diseases are among the most expensive disruptive and least well treated of human maladies arguably because they are not well understood Array tomography AT is a method for tissue imaging with resolution in all three dimensions sufficient to resolve individual synapses and provide quantitative characterization ...

DESCRIPTION provided by applicant One of the most complex forms of congenital heart disease hypoplastic left heart syndrome HLHS affects an average of in every newborns with a mortality rate of up to percent during the first year of life The overall goal f our research is to develop safe and effective ways to improve short term and long term survival rates in babies born with HL ...

ABSTRACT SUMMARY Pyruvate dehydrogenase complex PDC deficiency PDCD is a rare disease of mitochondrial energy failure in which the life of expectancy of affected children is severely truncated Treatment of PDCD remains a serious unmet challenge There has never been a controlled trial of any intervention for PDCD thus there is no proven therapy for affected patients Dichloroacetate DCA ...

PROJECT SUMMARY ABSTRACT Interactions between the gut microbiome and the immune system have been linked to inflammatory bowel disease IBD IBD is a chronic and progressive gastrointestinal disease characterized by uncontrolled activation of the intestinal immune system resulting in severe medical complications affecting over million patients in the United States It is a disease of high u ...

Summary In this fast track Phase I II STTR project a novel mass spectrometry method and a matched reagent kit will be developed for mapping modified nucleotides in human mitochondrial transfer RNA mt tRNA including the most abundant modification pseudouridine which hitherto has proved to be a technical challenge because it is an isomer of uridine These new research tools will be harnessed to build ...

DESCRIPTION provided by applicant There are currently no drugs or other therapeutic interventions that can reverse or halt the progression of Alzheimerandapos s disease AD Age is by far the greatest risk factor for AD and it is known from studies in both mice and humans that neuroinflammation is increased with old age and to an even greater extent in AD Therefore a drug that could reduce ...

DESCRIPTIONprovided by applicantThe purpose of this project is to develop an improvedminimally invasive treatment for intracranial aneurysmsIntracranial aneurysms are spherical outpouchings of blood vessels in the head that result from weakness in the vessel wallUnruptured aneurysms are present in approximatelyof the general populationand rupture can be devastating with a high morbidity and mortal ...

DESCRIPTION provided by applicant Genomic instability is a hallmark of cancer and represents a targetable vulnerability yet is underdeveloped as a therapeutic area The central goal of this Phase program is to complete the early preclinical development of a new class of new cancer therapeutics that uniquely target RAD delivering effective cancer cell selective therapy in subsets of bioma ...