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 kids needing stem cell transplants
Disease control CompletedThis study tested two different chemotherapy drugs (Treosulfan vs Busulfan) given before a stem cell transplant in 106 children with serious non-cancerous diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug …
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated May 26, 2026 07:55 UTC
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New registry tracks pregnancy in women with rare metabolic diseases
Knowledge-focused CompletedThis study gathered medical information from women with rare metabolism disorders (like methylmalonic acidemia) who were pregnant, planning pregnancy, or had been pregnant before. Researchers collected records from doctors and followed mothers and babies for one year after birth.…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 26, 2026 07:27 UTC
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Scientists hunt for missing genetic clues in rare diseases
Knowledge-focused CompletedThis study looked for new or overlooked genetic changes in people with inherited diseases, especially those whose standard genetic tests were unclear. Researchers examined DNA from 56 patients and their family members to find hidden variants that might explain their condition. Th…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC