PROPIONIC ACIDEMIA
Clinical trials for PROPIONIC ACIDEMIA explained in plain language.
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MRNA therapy takes on rare genetic disease: hope for propionic acidemia patients
Disease control Recruiting nowThis study tests an experimental mRNA medicine called mRNA-3927 in people with propionic acidemia, a rare inherited disorder that causes dangerous metabolic episodes. The treatment aims to help the body produce a missing enzyme, potentially reducing the frequency of these life-th…
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated May 26, 2026 06:34 UTC
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New mRNA therapy shows promise for rare metabolic disorder
Disease control Recruiting nowThis study looks at the long-term safety of an experimental medicine called mRNA-3927 for people with propionic acidemia, a rare genetic condition. Participants must have been in an earlier study of this drug. The goal is to see if the drug is safe over time and helps control the…
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated May 26, 2026 06:23 UTC
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Scientists track rare metabolic disease to uncover its secrets
Knowledge-focused Recruiting nowThis study follows people with propionic acidemia (PA), a rare metabolic disorder, to learn how the disease affects the body over years. Participants aged 2 and older visit the clinic annually for tests like blood draws, heart checks, and brain scans. The goal is to better unders…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated May 26, 2026 06:23 UTC
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New study aims to map rare disease without offering treatment
Knowledge-focused Recruiting nowThis study looks at medical records of 60 people with propionic acidemia, a rare genetic disorder. Researchers want to understand how the disease affects patients over time, including hospital visits and metabolic events. No new treatments are tested; the goal is to gather inform…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: ModernaTX, Inc. • Aim: Knowledge-focused
Last updated May 22, 2026 13:53 UTC