INBORN ERRORS OF METABOLISM
Clinical trials for INBORN ERRORS OF METABOLISM explained in plain language.
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Researchers seek 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 2275 participants over time using blood tests, imaging, and othe…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Apr 30, 2026 15:52 UTC
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Massive rare disease registry launches to unlock secrets of 30+ conditions
Knowledge-focused Recruiting nowThis study is creating a large database to collect information from 380 people with rare diseases like amyloidosis, sarcoidosis, and many others. Researchers will track survival, treatments, and side effects over time. The goal is to better understand these conditions and improve…
Matched conditions: INBORN ERRORS OF METABOLISM
Sponsor: Hospital Italiano de Buenos Aires • Aim: Knowledge-focused
Last updated Apr 28, 2026 13:02 UTC