Adenylosuccinic Acid Is a Non-Toxic Small Molecule In Vitro and In Vivo DOI Open Access
Cara A. Timpani,

Lorna Rasmussen,

Emma Rybalka

et al.

Published: Sept. 7, 2023

Adenylosuccinic acid (ASA) is a small molecule dicarboxylate that could be strong clinical development candidate for inherited myopathies involving dysregulated purine nucleotide metabo-lism. Currently, there are no published pharmacokinetic/dynamic or toxicology data available, albeit 10-year trial in Duchenne muscular dystrophy patients suggests chronically safe drug. In this study, we tested the toxicity of ASA to cultured myoblasts vitro and acute systemic mice. non-toxic with an LD50> 5000mg/kg. Some background necrotic foci liver, kidney gastrointestinal tract were shown likely incidental but warrant follow-up sub-/chronic oral exposure studies.

Language: Английский

Adenylosuccinic Acid Is a Non-Toxic Small Molecule In Vitro and In Vivo DOI Open Access
Cara A. Timpani,

Lorna Rasmussen,

Emma Rybalka

et al.

Published: Sept. 7, 2023

Adenylosuccinic acid (ASA) is a small molecule dicarboxylate that could be strong clinical development candidate for inherited myopathies involving dysregulated purine nucleotide metabo-lism. Currently, there are no published pharmacokinetic/dynamic or toxicology data available, albeit 10-year trial in Duchenne muscular dystrophy patients suggests chronically safe drug. In this study, we tested the toxicity of ASA to cultured myoblasts vitro and acute systemic mice. non-toxic with an LD50> 5000mg/kg. Some background necrotic foci liver, kidney gastrointestinal tract were shown likely incidental but warrant follow-up sub-/chronic oral exposure studies.

Language: Английский

Citations

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