INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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New immune cell boost may shield kids from Post-Transplant infections
Disease control Recruiting nowThis study tests whether adding memory immune cells (CD45RO) to a stem cell transplant can help children fight off dangerous viral and fungal infections after the procedure. The transplant uses donor stem cells that have been stripped of certain cells to prevent graft-versus-host…
Matched conditions: INBORN ERRORS OF METABOLISM
Phase: PHASE1, PHASE2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 18, 2026 11:50 UTC
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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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Scientists launch deep dive into rare metabolic disease MMA
Knowledge-focused Recruiting nowThis study follows people with methylmalonic acidemia (MMA) and related disorders to learn how the disease progresses over time. Researchers will collect medical history, perform tests like blood draws and MRIs, and look for new genetic causes. The goal is to better understand co…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jun 23, 2026 11:57 UTC
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New registry aims to unlock secrets of rare diseases
Knowledge-focused Recruiting nowThis study is creating a large database to collect information on people with rare diseases like amyloidosis, sarcoidosis, and Gaucher disease. Researchers will track patients' health over time, including their symptoms, treatments, and outcomes. The goal is to improve diagnosis …
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated Jun 22, 2026 11:55 UTC