INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New hope for kids needing transplants: gentler prep drugs tested
Disease control CompletedThis study aimed to find a safer and effective drug treatment for children needing a stem cell transplant for serious non-cancerous diseases like immune deficiencies and metabolic disorders. It directly compared two different drug combinations used to prepare a child's body to re…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated Mar 19, 2026 14:56 UTC
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Tracking rare pregnancies: how metabolic disorders affect moms and babies
Knowledge-focused CompletedThis study created a registry to collect health information from women with rare inherited metabolic disorders who are pregnant or planning pregnancy. Researchers gathered medical records to understand how these conditions affect both the mother's health during pregnancy and the …
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:32 UTC
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Scientists hunt for hidden genetic clues in rare diseases
Knowledge-focused CompletedThis study aimed to discover new genetic changes that cause inherited disorders, especially in patients where standard genetic testing was inconclusive. Researchers analyzed DNA and RNA from blood samples of 56 participants, including patients and their family members. The goal w…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC