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Palynziq and PKU: Treatment Impacts on Diet Quality, Neurological Health, Nutritional Status, and the Metabolome
Phenylketonuria (PKU) is an inherited metabolic disorder that impairs the metabolism of the essential amino acid phenylalanine (Phe). Without stringent dietary…
Phenylketonurias
Emory UniversityNCT04404530
Observational
Development of Novel Psychological Assessment Tools and Anxiety Intervention for PKU: Intervention Trial
While previous PKU intervention research has largely focused on pharmacological treatment of elevated Phe levels, the adaptation of evidence-based psychosocial…
Phenylketonurias
University of Missouri-ColumbiaNCT06560736
Phase 1
An Open-Label, Multiple-Center, Phase I/II Dose Escalation Study for the Safety and Efficacy of NGGT002 in Adults With Classic Phenylketonuria
This is a Phase 1/2, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002 in adult subjects w…
Phenylketonurias
NGGT INC.NCT06332807