INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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Immune cell boost may shield kids from Post-Transplant infections
Disease control Recruiting nowThis study tests whether adding a special type of immune cell (memory T cells) after a donor stem cell transplant can help prevent serious viral and fungal infections. It involves up to 100 children and young adults with blood cancers or immune disorders. The approach aims to spe…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New hope for kids with rare blood diseases: expanded stem cell access
Disease control Recruiting nowThis program provides stem cell transplants for up to 100 children and young adults with life-threatening blood disorders, such as leukemia or immune deficiencies, who do not have a matched sibling donor. The approach uses a special technique to remove certain immune cells from t…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 11, 2026 20:46 UTC
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New registry aims to shed light on rare diseases
Knowledge-focused Recruiting nowThis study is creating a large database to collect information about people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will track participants' health, treatments, and outcomes over time to better understand these conditions. The goal is to imp…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated May 11, 2026 20:49 UTC
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NIH launches major study to unlock mysteries of rare metabolic disease
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 oth…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 07, 2026 18:42 UTC