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Vosoritide
Vosoritide (INN, codenamed BMN-111) is an experimental drug for the treatment of achondroplasia, the most common cause of dwarfism. It is being developed by BioMarin Pharmaceutical and, being the only available causal treatment for this condition, has orphan drug status in the US as well as Europe. , it is in Phase II clinical trials.
==Mechanism of action==
Fibroblast growth factor receptor 3 (FGFR3) is a receptor that normally down-regulates cartilage and bone growth when activated by one of the proteins known as acidic and basic fibroblast growth factor. It does so by inhibiting the development (cell proliferation and differentiation) of chondrocytes, the cells that produce and maintain the cartilaginous matrix which is also necessary for bone growth. Children with achondroplasia have one of several possible FGFR3 mutations resulting in constitutional (permanent) activity of this receptor, resulting in overall reduced chondrocyte activity and thus bone growth.
The protein C-type natriuretic peptide (CNP), naturally found in humans, reduces the effects of over-active FGFR3. Vosoritide is a CNP analogue with the same effect but prolonged half-life,〔 allowing for once-daily administration.〔
抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』
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