Award Data

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 ...

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 ...

DESCRIPTION provided by applicant Gene transfer for the treatment of cardiovascular diseases is bedeviled by inability to obtain safely and easily sufficient cardiac transgene expression Current methods of gene transfer for heart disease include intramuscular injection into heart muscle or intracoronary delivery approaches that are cumbersome to apply Consequently we have considered the u ...

DESCRIPTION provided by applicant Atopic dermatitis AD or severe eczema is a very common life long skin disease with of adults in the US requiring systemic therapy Clinical manifestations of AD include multiple inflamed lesions erosions accompanied by lichenification thick leathery skin fibrotic papules and severely dry skin with increased susceptibility to infection A major uncont ...

DESCRIPTIONprovided by applicantThe goal of this Fast Track STTR project is to develop a novel CardioPortTM surgical device that will enable minimally invasive beating heart repair without use of cardiopulmonary bypassCPBThere is a critical unmet need for new instruments to improve the surgical repair of heart defects in childrenCurrent best surgical practices involve either open heart surgery wit ...

DESCRIPTION provided by applicant Overall Staphylococcus aureus is a gram positive bacteria which possesses a multitude of virulence factors It is a frequent and severe pathogen in hospitals and of increasing concern in the community where it results in severe skin infections pneumonia bacterial endocarditis and sepsis A significant proportion of these infections are the result of methici ...