SFX-01 is therapeutic against myeloproliferative disorders caused by activating mutations in Shp2

SFX-01 is therapeutic against myeloproliferative disorders caused by activating mutations in Shp2

Abstract Activating mutations of Src homology-2 domain-containing protein tyrosine phosphatase-2 (Shp2) cause multiple childhood conditions for which there is an unmet therapeutic need, including juvenile myelomonocytic leukemia (JMML) and Noonan syndrome. SFX-01, an α-cyclodextrin-stabilized sulfor...

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Main Authors: Hyun-Ju Cho, Joy Smith, Christopher H Switzer, Eleni Louka, Rebecca L Charles, Oleksandra Prysyazhna, Ewald Schroder, Mariana Fernandez-Caggiano, Daniel Simoes de Jesus, Seda Eminaga, Xiaoke Yin, Xiaoping Yang, Steven Lynham, Manuel Mayr, Valle Morales, Katiuscia Bianchi, Vinothini Rajeeve, Pedro R Cutillas, Adam J Mead, Philip Eaton
Format: Article
Language:English
Published: Springer Nature 2025-07-01
Series:EMBO Molecular Medicine
Subjects:
Shp2
SFX-01
Sulforaphane
Noonan Syndrome
Myeloproliferative Disorders
Online Access:https://doi.org/10.1038/s44321-025-00267-7
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