GH1 and Severe short stature: Growth hormone therapy has been used to treat dwarfism but is clarified to be ineffective for height gain in most congenital skeletal dysplasia, and in some cases with severe spinal deformities, it even results in worsened kyphosis and lordosis (Kanazawa et al., 2003), emphasizing the need for a deep understanding of the molecular pathogenesis for effective treatment development.