INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
Never miss a new study
Get alerted when new INBORN ERRORS OF METABOLISM trials appear
Sign up with your email to follow new studies for INBORN ERRORS OF METABOLISM, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
New drug combo may make stem cell transplants safer for kids with rare diseases
Disease control CompletedThis study tested two different drug combinations given before a stem cell transplant in 106 children with serious non-cancer diseases like immune deficiencies or blood disorders. The goal was to see which drug (treosulfan or busulfan) leads to fewer deaths from transplant compli…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated May 17, 2026 01:30 UTC
-
Hunting for hidden genetic clues in rare diseases
Knowledge-focused CompletedThis study looked for new genetic changes that might cause inherited diseases, especially in people who already have a genetic disorder but whose genetic tests were not clear. Researchers examined DNA from 56 participants, including patients and their unaffected family members. T…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 11, 2026 20:40 UTC
-
New registry tracks rare metabolic disorders in pregnancy
Knowledge-focused CompletedThis study gathered information from the 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. No extra tests or treatments w…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 11, 2026 20:39 UTC