PROPIONIC ACIDEMIA
Clinical trials for PROPIONIC ACIDEMIA explained in plain language.
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Experimental gene therapy targets rare metabolic disease in toddlers
Disease control Recruiting nowThis early-stage trial tests a gene therapy for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises and organ damage. The study will enroll 9 children aged 6 months to 2 years with confirmed PCCA gene mutations. The therapy uses a harmless virus to …
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1 • Sponsor: Mayo Clinic • Aim: Disease control
Last updated Jun 27, 2026 14:02 UTC
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New mRNA treatment for rare metabolic disease moves to Long-Term safety check
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 disorder that prevents the body from breaking down certain proteins and fats. About 50 participants who were in an earlier study will continue …
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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MRNA therapy takes on rare metabolic disease
Disease control Recruiting nowThis study tests an experimental mRNA therapy called mRNA-3927 for propionic acidemia, a rare genetic disorder that causes dangerous metabolic crises. The trial involves about 77 participants of all ages, starting with older children and adults. Researchers will check safety, fin…
Matched conditions: PROPIONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Scientists launch Largest-Ever study to unravel rare metabolic disease
Knowledge-focused Recruiting nowThis study follows people with propionic acidemia (PA), a rare metabolic disorder, to track how the disease affects the body over time. Researchers will collect medical history, blood, urine, and other samples, and perform imaging and heart tests during annual hospital visits. Th…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: National Human Genome Research Institute (NHGRI) • Aim: Knowledge-focused
Last updated Jul 02, 2026 00:00 UTC
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No treatment, just data: new study tracks rare metabolic disease
Knowledge-focused Recruiting nowThis study is not testing any new drug or treatment. Instead, researchers will look back at medical records of 60 people with propionic acidemia, a rare genetic disorder that affects how the body breaks down certain proteins and fats. The goal is to better understand the disease,…
Matched conditions: PROPIONIC ACIDEMIA
Sponsor: ModernaTX, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:02 UTC