Pearson syndrome
MONDO:0010797Pearson syndrome is characterized by refractory sideroblastic anemia, vacuolization of bone marrow precursors and exocrine pancreatic dysfunction.
Also known as: Pearson marrow-pancreas syndrome, Pearson's marrow/pancreas syndrome, Pearson's syndrome, sideroblastic Anaemia with marrow cell vacuolization and exocrine pancreatic dysfunction, sideroblastic Anemia with marrow cell vacuolization and exocrine pancreatic dysfunction, sideroblastic anaemia with marrow cell vacuolization and exocrine pancreatic dysfunction (formerly), sideroblastic anemia with marrow cell vacuolization and exocrine pancreatic dysfunction (formerly)
3 clinical trials for this condition and its sub-types.
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Massive study aims to uncover cancer secrets in rare bone marrow diseases
Knowledge-focused Recruiting nowThis natural history study follows up to 4,000 people with inherited bone marrow failure syndromes (IBMFS) and their families to learn why they are prone to certain cancers. Researchers will track health over time, collect genetic samples, and look for clues that separate those w…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Sep 09, 2026 17:00 UTC
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Worldwide data pool could pave the way for mitochondrial disease trials
Knowledge-focused Recruiting nowThis study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …
Sponsor: LMU Klinikum • Aim: Knowledge-focused
Last updated Aug 09, 2026 00:00 UTC