INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New hope for kids needing Life-Saving transplants
Disease control Recruiting nowThis trial provides expanded access to stem cell transplants for children with serious blood, immune, or metabolic diseases who lack a perfectly matched sibling donor. It uses a special device to remove certain cells from the donor's blood to reduce the risk of a dangerous compli…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Landmark study seeks to unlock mysteries of rare metabolic disease
Knowledge-focused Recruiting nowThis study aims to better understand methylmalonic acidemia (MMA), a rare and serious inherited metabolic disorder. Researchers will observe over 2,000 patients to learn how the disease progresses, identify complications, and find biological markers. The goal is to gather essenti…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Mar 11, 2026 14:54 UTC
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Hospital launches major effort to map the mysteries of rare diseases
Knowledge-focused Recruiting nowThis study aims to create a single, large registry to collect information on many different rare diseases. It will enroll about 380 patients already being treated at the Hospital Italiano de Buenos Aires who have one of the listed rare conditions. The goal is to gather data to be…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated Mar 10, 2026 12:53 UTC