INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New drug combo may make stem cell transplants safer for kids with blood disorders
Disease control CompletedThis study tested two different drug combinations given before a stem cell transplant in 106 children with non-cancerous diseases like immune deficiencies and blood disorders. The goal was to see if one drug (treosulfan) caused fewer serious side effects or deaths than the standa…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE2 • Sponsor: medac GmbH • Aim: Disease control
Last updated May 01, 2026 15:59 UTC
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Scientists hunt for missing genetic clues in mysterious diseases
Knowledge-focused CompletedThis study looked for new genetic changes that might explain diseases in people who already have a genetic disorder but unclear genetic test results. Researchers studied 56 participants, including patients and their unaffected family members. The goal was to find missing genetic …
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 01, 2026 16:00 UTC