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Gene therapy for long-term treatment of GSDIa
Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…
Automatic segmentation software to diagnose retinopathy of prematurity (ROP) from optical coherence tomography images in preterm infants
Unmet Need Retinopathy of prematurity (ROP) is a developmental vascular proliferative disorder that occurs in approximately 50-70% of preterm infants and is a leading cause of childhood blindness. The most critical factor in identifying ROP…
Aptamer anticoagulant therapy to reduce complications of ECMO treatment
Unmet Need Extracorporeal membrane oxygenation (ECMO) is a life-saving treatment for critically ill patients with low oxygen levels. During ECMO, the patient’s blood flows through an external oxygenation device. This requires co-treatment with an anticoagulant…
The Trail Guide curriculum for childhood obesity treatment program model
Unmet Need Childhood obesity is an ongoing health concern involving cardiometabolic risk, reduced quality of life and long-term health consequences. Current child obesity treatment guidelines recommend 26 or more hours of behavior treatment, delivered over…
A method of screening and monitoring glycogen storage diseases with a single biomarker
Unmet Need Glycogen storage disease type I (GSD I) is an autosomal recessive inherited disorder of carbohydrate metabolism associated with life threatening fasting hypoglycemia, and significant morbidity and mortality. Currently, the standard of care therapy…