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Protego Biopharma's Myelofibrosis Program is a discovery-stage research initiative focused on the development of small molecule pharmacological chaperones. These molecules are designed to correct or modulate protein misfolding, a process central to the pathogenesis of myelofibrosis and various protein amyloidopathies. By stabilizing the native conformation of specific proteins, the program aims to prevent the formation of toxic aggregates and restore cellular homeostasis. This approach is part of Protego's broader platform targeting protein misfolding diseases, which also includes programs for AL amyloidosis and liver diseases.
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