Award Data

Abstract Aggregation of mutated myocilin (MYOC), a protein found in the endoplasmic reticulum (ER) of trabecular meshwork (TM) cells, disrupts the outflow of aqueous humor from the eye, causing increase in intraocular pressure (IOP), progressive retinal ganglion cell (RGC) death, and degeneration of the optic nerve, leading to primary open-angle glaucoma (POAG), characterized by irreversible visio ...

ABSTRACT / PROJECT SUMMARY Wet age-related macular degeneration (AMD) is the leading cause of irreversible blindness in the developed world. Anti-VEGF therapy is currently the gold standard for wet AMD treatment. However, up to 50% of patients in the long term have a suboptimal response to anti-VEGF therapy. A better alternative method for treatment of AMD is urgently needed. We have recently inve ...

Abstract Tuberculosis (TB), caused by Mycobacterium tuberculosis (Mtb), is the biggest killer among infectious diseases. TB is also responsible for a quarter of all deaths associated with Antimicrobial Resistance (AMR). It is projected that by 2050, 75 million people, or one person every 12 seconds, will die due to AMR associated with TB. Long treatment times and increasing resistance to TB drugs ...

DESCRIPTION provided by applicant The pursuit of novel therapies informed by the astounding advances in biomedical science of the past half century has been fruitful for many disorders However inherited muscle diseases such as muscular dystrophy remain stubbornly resistant to sophisticated molecular approaches to therapy A few therapies are finally edging towards approval but it has become ...

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 ...

ABSTRACT: Cutaneous vascular malformations (CVM), such as port wine stain (PWS) which occurs in 0.3–0.5% of newborns, are often a cause of great concern to patients for both medical and cosmetic reasons. Laser irradiation with flashlamp-pumped pulsed dye lasers, i.e. photothermolysis therapy (PTT), is currently the gold standard treatment for PWS. Most patients with PWS, however, fail to clear c ...

Obstetricians measure fetal heartrate (FHR) and uterine contractions to evaluate fetal health during pregnancy and labor. This monitoring is performed to decrease the development of complications while minimizing the need for unnecessary obstetric interventions and is especially important for women with high-risk pregnancies. However, many women, especially in rural areas, do not have the time or ...

Project Abstract An estimated 5.7 million Americans have Alzheimer’s disease or a related dementia (ADRD) and one in ten people age 65 or older show symptoms of Alzheimer’s disease. As ADRD progresses so does the level of dependency on others. As the disease progresses, higher levels of dependence in people with Alzheimer’s disease are associated with significantly increased burden and costs ...

EXECUTIVE SUMMARY - huESC HTS2 - human Toxic stress effects in 1st trimester pregnancy leads to $10s of billions of US health care costs/year and is a significant part of the $2.5-4B/year US Developmental and Reproductive Technology (DART industry. Current DART techniques use slow, expensive pregnant rodent tests, or tests of embryonic stem cells (ESCs) that are cultured to lose “stemness” and ...

ABSTRACT Traditional approaches to drug development for Alzheimer s disease are becoming increasingly expensive and in many cases disappointingly unsuccessful Based on preliminary in vitro and in vivo studies we have identified a novel small molecule methyl dimethyl oxo dihydrobenzo c naphthyridine carboxylate UK that significantly decreases A production and pro in ...