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Open access Oct 2026

Safety and pharmacokinetics of inhaled aloxistatin: A first-in-human phase 1 dose escalation trial.

AIMS Aloxistatin (E64d) is a membrane-permeable cysteine protease inhibitor prodrug that is converted by esterases to the active metabolite E64c (loxistatin). This first-in-human study evaluated the safety, tolerability and pharmacokinetics of single ascending doses of inhaled E64d in healthy volunteers. METHODS In t...

S. Fähndrich, Franziska Friedrich, Daniel Soriano et al. · 0 citations
Open access Aug 2026

Irritable Bowel Disease: Think Malonic and Methylmalonic Aciduria (CMAMMA)!

Combined malonic and methylmalonic aciduria (CMAMMA; OMIM 614265) is a rare disorder of mitochondrial fatty acid synthesis caused by pathogenic variants in the ACSF3 gene, which encodes malonyl‐CoA synthetase. The clinical presentation is highly heterogeneous and may include seizures, memory impairment, psychiatric man...

Khaled I Alatibi, M. J. Hug, Sara Tucci · 0 citations

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