METHYLMALONIC ACIDEMIA
Clinical trials for METHYLMALONIC ACIDEMIA explained in plain language.
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New mRNA therapy for rare metabolic disease moves to Long-Term safety check
Disease control Recruiting nowThis study looks at the long-term safety and effects of an experimental mRNA medicine called mRNA-3705 for people with a rare genetic disorder called methylmalonic acidemia (MMA). Participants have already taken part in earlier studies of this drug. The goal is to see if the trea…
Matched conditions: METHYLMALONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated May 14, 2026 12:03 UTC
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NIH launches major 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 can cause serious health problems like kidney failure, strokes, and developmental delays. Researchers will observe up to 2,275 participants over time using blood tests, imaging, and oth…
Matched conditions: METHYLMALONIC ACIDEMIA
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 07, 2026 18:42 UTC