INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New cell addback aims to slash infection risk in child stem cell recipients
Disease control Recruiting nowThis study tests whether adding a specific type of immune cell (memory T cells) after a stem cell transplant can help prevent serious viral and fungal infections. About 100 children and young adults with blood cancers, immune disorders, or bone marrow failure will receive this ex…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 26, 2026 11:40 UTC
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New stem cell transplant option for kids without a perfect donor match
Disease control Recruiting nowThis program provides stem cell transplants for up to 100 children and young adults with life-threatening blood or immune system diseases who do not have a perfectly matched sibling donor. The transplant uses a special technique to remove certain immune cells from the donated ste…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: EARLY_PHASE1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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NIH launches Long-Term 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 affects how the body processes certain proteins and fats. Researchers will observe up to 2,275 participants over time, using tests like blood draws, imaging, and developmental assessmen…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 26, 2026 11:32 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 patients' health, treatments, and outcomes over time. The goal is to better understand these conditions and improv…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated May 26, 2026 11:31 UTC