PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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New gene therapy aims to tame PKU in first human test
Disease control OngoingThis early-phase study tests a single IV dose of NGGT002 gene therapy in 15 adults with phenylketonuria (PKU), a genetic disorder that causes harmful buildup of phenylalanine. The main goal is to check safety over one year, with long-term follow-up for five years. Researchers wil…
Matched conditions: PHENYLKETONURIAS
Phase: EARLY_PHASE1 • Sponsor: The First Affiliated Hospital of Bengbu Medical University • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: PHENYLKETONURIAS
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC