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Preclinical studies to validate the efficacy of novel mechanism-of-action small molecule inhibitors to treat Duchenne muscular dystrophySBC: RIDGELINE THERAPEUTICS LLC Topic: NIAMS
Progressive muscle weakness and degeneration is a hallmark of Duchenne muscular dystrophyDMDIn DMD patientsthe lack of dystrophin reduces muscle fiber structural integritymaking muscles vulnerable to persistent injury and damageRepairing these damaged muscles requires the continual activation of muscle stem cellsmuSCwhich leads to muSC dysfunction and senescenceand ultimately the muscle degenerati ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: Oncoimmune Inc Topic: 100
Project Summary AbstractThe recent rapid expansion of genomic data greatly contributes to the understanding of disease development and progressionTo translate these genetic discoveries into clinical applicationswe need to develop therapeutic platforms that can specifically modulate the expression of disease related genes in vivoSmall RNAssuch as microRNAs and siRNAs function in messenger RNA silen ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: ABRI SCIENCE, LLC Topic: 300
PhenylketonuriaPKUis a genetic disorder of metabolism characterized by a pathological elevation of phenylalaninePhein the body stemming from a dysfunctional phenylalanine hydroxylase enzymePAHThe resulting high concentrations of Phexcan result in severe intellectual disabilitybehavioral problemsand neurological deficitse gseizuresStrict control of dietary Phe is the primary treatment for PKU curre ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: CORD BLOOD PLUS, INC. Topic: 102
PROJECT SUMMARY ABSTRACT The objective of this proposal is to move our product forward toward commercialization by improving the quality of life for breast cancer chemotherapy patientsThis will be achieved by the following Specific AimsAimwill optimize differentiation of GMPs and neutrophil lineages in culture in response to G CSFThis will be assessed by flow cytometry to quantify production of ne ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: Data2Care Technologies, LLC Topic: NICHD
Project Summary Abstract Acute gastroenteritisAGEinfectious diarrhea is a common condition that is costly in the US and highmortality in developing nationsThe cornerstone of AGE management is oral rehydration therapyORTan evidence based technique that has saved overmillion lives in theyears since its introductionThe problem we aim to address is ORT s underuse in developed countries specifically in ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: CLEARNANO INC Topic: 400
SummarySystemic lupus erythematosusSLEor lupus is an autoimmune disease that impacts more thanmillion people worldwideincludingmillion AmericansNearlyof these patients will develop lupus nephritiswhich can cause kidney failureAs a resultits financial burden to individual patient can reach $yearmuch higher than many other diseases because of kidney transplantationAlthough early treatment of lupus n ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: LYST THERAPEUTICS LLC Topic: NHLBI
SUMMARY Despite significant advances in the surgical and medical management of congenital heart diseasecongenital cardiac anomalies remain a leading cause of death in the newborn periodMost severe forms of congenital heart disease require multiplestaged surgical interventionsA significant source of the heightened morbidity and mortality associated with these operations is the development of cardia ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
The Daily Progress System- A Recovery Support Tool to Reduce Health Disparities in Outpatient Substance Use TreatmentSBC: COG ANALYTICS, LLC Topic: 102
ABSTRACTSubstance use disordersSUDsincluding opioid use disorderscontinue to be one of the most serious public health issues in the USleading to negative physical and mental health problemsfamily disruptionand premature deathAfrican Americans with drug use histories that are under criminal justice supervision are particularly vulnerable to experiencing the negative consequences associated with SUD ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: KLEIN BUENDEL, INC. Topic: 102
Project Summary While studies support the efficacy of comprehensiveschool wide interventions in reducing bullyingthese types of programs can require significant time and financial resources for implementationresulting in barriers to providing school based bullying preventionespecially in low income and rural communitiesAdditionallyalthough training bystanders to act asdefenderson behalf of targets ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health
SBC: Vova Ida, LLC Topic: 999
SUMMARYParkinsonandapos s diseasePDis the second most prevalent neurodegenerative disorder after Alzheimerandapos s diseaseADaffecting more thanmillion people in the United StatesBecause age is the major risk factorthe prevalence of both diseases is steadily increasing as our population agesPD belongs to the group of protein misfolding neurodegenerative diseases that are due to the misfolding and ...STTR Phase I 2019 Department of Health and Human ServicesNational Institutes of Health