PROPIONIC ACIDEMIA
Clinical trials for PROPIONIC ACIDEMIA explained in plain language.
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Moderna tests mRNA therapy for rare, devastating childhood disease
Disease control Recruiting nowThis study is testing an investigational mRNA therapy called mRNA-3927 for people with propionic acidemia (PA), a rare and serious genetic disorder. The goal is to find a safe and effective dose that can reduce dangerous metabolic crises and improve health. The study will enroll …
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:07 UTC
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Moderna tests Long-Term safety of rare disease treatment
Disease control Recruiting nowThis study follows people with propionic acidemia who were in an earlier trial of the experimental treatment mRNA-3927. It aims to check the long-term safety of continuing this treatment and see if it helps control the disease. About 50 participants will receive the same dose the…
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Mar 06, 2026 15:37 UTC
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Scientists launch major study to unlock mysteries of rare metabolic disease
Knowledge-focused Recruiting nowThis study aims to better understand propionic acidemia, a rare genetic disorder where the body cannot properly break down certain parts of food. Researchers will observe over 1,000 participants, including people with the condition and some family members, over several years thro…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:28 UTC
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Scientists dig into medical records to map rare disease journey
Knowledge-focused Recruiting nowThis study aims to learn more about propionic acidemia, a rare genetic disorder, by looking back at the medical records of 60 patients. Researchers will collect information about how often patients experience serious health events called metabolic decompensations and related hosp…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: ModernaTX, Inc. • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC