METHYLMALONIC ACIDEMIA
Clinical trials for METHYLMALONIC ACIDEMIA explained in plain language.
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Moderna tests mRNA shot to fight rare, Life-Threatening metabolic disease
Disease control OngoingThis study is testing an experimental mRNA therapy called mRNA-3705 for people with a rare, inherited metabolic disorder called methylmalonic acidemia (MMA). The main goals are to see if the treatment is safe and if it can lower the levels of harmful acids that build up in the bl…
Matched conditions: METHYLMALONIC ACIDEMIA
Phase: PHASE1, PHASE2 • Sponsor: ModernaTX, Inc. • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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Scientists track patients for 15 years after rare disease gene therapy
Disease control OngoingThis study is monitoring the long-term safety of four patients who previously received an experimental gene therapy (hLB-001) for a rare genetic disorder called methylmalonic acidemia (MMA). The main goal is to check for any side effects that may appear over a very long period—up…
Matched conditions: METHYLMALONIC ACIDEMIA
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC