Congenital, hereditary, and neonatal diseases and abnormalities
Clinical trials for Congenital, hereditary, and neonatal diseases and abnormalities explained in plain language.
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Could a stem cell shot before birth fix fanconi anemia?
Disease control Not yet recruitingThis early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant wo…
Matched conditions: CONGENITAL, HEREDITARY, AND NEONATAL DISEASES AND ABNORMALITIES
Phase 1/2 • Sponsor: Agnieszka Czechowicz • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Can tiny tablets replace protein powder for kids with PKU?
Symptom relief Not yet recruitingThis trial tests whether XPhe Piccos, a slow-release protein substitute in tiny tablets without phenylalanine, is acceptable and well-tolerated in children aged 3 and older with phenylketonuria (PKU) or hyperphenylalaninemia. Participants replace some or all of their usual protei…
Matched conditions: CONGENITAL, HEREDITARY, AND NEONATAL DISEASES AND ABNORMALITIES
Sponsor: metaX Institut fuer Diatetik GmbH • Aim: Symptom relief
Last updated Sep 05, 2026 00:00 UTC