MDBR - Fibrous Dysplasia/McCune Albright Syndrome

Two grants available, each for up to $53,614. Fibrous dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease caused by somatic mutations in GNAS. The mutation results in constitutive activation of the signaling protein, Gsα, and downstream cAMP signaling. Skeletal manifestations include bone pain, fractures, deformity, and osteomalacia/rickets. Any study that focuses on the pathogenesis of FD/MAS, or clinical investigative studies to address any of the unmet needs in FD/MAS patients and their management will be considered. Research priorities for the Fibrous Dysplasia Foundation include: generation of new mouse models to study FD/MAS; studies to understand the mechanism and/or treatment of FD-related bone pain; development/testing of therapeutics, especially those targeting Gsα, PKA or Wnt signaling pathways, including through the use of oligonucleotides; or studies of the molecular etiology, especially the role of RANKL, IL6, cAMP and FGF23. These grants are made possible by Team FD and the Fibrous Dysplasia Foundation.

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