@entity21695 Analog as Therapy for XXXX .

@entity1 @entity1610 @entity21695 @entity2557 @entity5884 @entity19 @entity31738 @entity4149 @entity72863
@entity2557 ( @entity2557 ) is an important regulator of bone formation. Gain-of-function mutations in the @entity2557 gene result in @entity1610 which include @entity5884 ( @entity5884 ), the most common form of dwarfism, in which skull, appendicular and axial skeletons are affected. The skeletal phenotype of @entity1 with @entity5884 showed defective proliferation and differentiation of the chondrocytes in the growth plate cartilage. Both endochondral and membranous ossification processes are disrupted during development. At cellular level, @entity2557 mutations induce increased phosphorylation of the @entity4149 kinase receptor @entity2557 , which correlate with an enhanced activation of its downstream signaling pathways. Potential therapeutic strategies have emerged for @entity5884 . Several preclinical studies have been conducted such as the @entity21695 ( @entity21695 ) analog ( @entity72863 ), intermittent parathyroid hormone injections, soluble @entity2557 therapy, and @entity31738 and statin treatments. Among the putative targets to antagonize @entity2557 signaling, @entity21695 (or @entity72863 ) is one of the most promising strategies. @entity72863 acts as a key regulator of longitudinal bone growth by downregulating the mitogen-activated protein kinase pathway, which is activated as a result of a @entity2557 gain-of-function mutation. Preclinical studies showed that @entity72863 treatment led to a large improvement in skeletal parameters in Fgfr3Y367C/+ @entity19 mimicking @entity5884 . In 2014, a clinical trial (phase 2) of @entity72863 in pediatric @entity1 with @entity5884 has started. This first clinical trial marks the first big step towards real treatment for these @entity1 .
