Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia - Nature
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from Nature11 months ago
No dose-limiting toxicities occurred during the mRNA-3927 therapy in participants with propionic acidaemia.
Naturehttps://www.nature.com/articles/s41586-024-07266-7?error=cookies_not_supported&code=1fa17bd1-f732-4ff8-8064-c3db0a7f42f2
Preliminary analysis showed a 70% reduction in the risk of metabolic decompensation events with mRNA-3927 therapy.
Naturehttps://www.nature.com/articles/s41586-024-07266-7?error=cookies_not_supported&code=1fa17bd1-f732-4ff8-8064-c3db0a7f42f2
Read at Nature
#propionic-acidemia #mrna-therapy #clinical-trial #metabolic-disorders
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