INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New chemo combo may lower death risk in child stem cell transplants
Disease control CompletedThis study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated Jun 17, 2026 12:03 UTC
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New registry tracks rare metabolic disorders in pregnancy
Knowledge-focused CompletedThis study gathered information from medical records of women with rare metabolic conditions (inborn errors of metabolism) who were pregnant or planning pregnancy. Researchers aimed to understand how these conditions affect both mother and baby during and after pregnancy. No extr…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jun 22, 2026 12:09 UTC
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Scientists hunt for missing genetic clues in mystery diseases
Knowledge-focused CompletedThis study aimed to find new genetic changes that might cause inherited diseases, especially in people who already have a diagnosis but whose genetic tests were not clear. Researchers studied 56 patients and their family members to look for hidden variants in parts of the DNA tha…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 17, 2026 12:09 UTC