METHYLMALONIC ACIDEMIA
Clinical trials for METHYLMALONIC ACIDEMIA explained in plain language.
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Hope for rare disease patients: long-term treatment study continues
Disease control Recruiting nowThis study continues testing mRNA-3705 in people with methylmalonic acidemia (MMA), a rare genetic disorder. It follows patients who previously received this experimental treatment in earlier studies to check long-term safety and effectiveness. The goal is to see if continued tre…
Matched conditions: METHYLMALONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC
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Landmark study seeks to unlock mysteries of rare metabolic disease
Knowledge-focused Recruiting nowThis study aims to better understand methylmalonic acidemia (MMA), a rare and serious inherited metabolic disorder. Researchers will observe over 2,000 patients to learn how the disease progresses, identify complications, and find biological markers. The goal is to gather essenti…
Matched conditions: METHYLMALONIC ACIDEMIA
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Mar 11, 2026 14:54 UTC