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 02:39 UTC
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Rare disease pregnancy registry seeks answers for moms and babies
Knowledge-focused CompletedThis study collected medical records from women with inborn errors of metabolism (conditions that affect how the body turns food into energy) who were pregnant or planning pregnancy. Researchers tracked their health and their baby's development for one year after birth. The goal …
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jun 15, 2026 19:04 UTC
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Scientists hunt for missing genetic clues in mysterious inherited diseases
Knowledge-focused CompletedThis study looked for new genetic changes in people with inherited diseases whose standard genetic tests were not clear. Researchers examined DNA from 56 participants, including patients and their unaffected family members. The goal was to find hidden variants that might explain …
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 02, 2026 11:57 UTC