INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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Lifesaving stem cell transplant now available for more patients
Disease control Recruiting nowThis program provides stem cell transplants to patients with serious blood disorders, such as leukemia or immune deficiencies, who do not have a fully matched sibling donor. It uses a special technique to remove certain immune cells from the donated stem cells to reduce the risk …
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 15, 2026 19:09 UTC
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New cell addback may shield kids from deadly infections after transplant
Disease control Recruiting nowThis study tests whether adding a special type of immune cell (memory T cells) after a stem cell transplant can help children fight off viral and fungal infections. About 100 children and young adults with blood cancers, immune disorders, or bone marrow failure will receive this …
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 15, 2026 19:04 UTC
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Scientists track rare metabolic disease to unlock its secrets
Knowledge-focused Recruiting nowThis study aims to learn more about methylmalonic acidemia (MMA), a rare genetic disorder that can cause serious health problems like kidney failure, strokes, and developmental delays. Researchers will observe up to 2,275 participants over time, using blood tests, imaging, and ot…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jun 08, 2026 13:09 UTC
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Rare disease database aims to unlock clues for 380 patients
Knowledge-focused Recruiting nowThis study is creating a detailed registry of people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will collect information from medical records and patient reports to learn how these diseases progress, how they are treated, and what outcomes pati…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated Jun 08, 2026 13:04 UTC