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Publication of impact on health-related quality of life from 3 year data with vosoritide. These data suggest that after 3 years of treatment, vosoritide demonstrates a positive effect on physical…
Achondroplasia is the most common skeletal dysplasia and is characterized by a variety of medical, functional and psychosocial challenges across the lifespan. There is variability in the clinical care pathways…
Despite the recognized expertise of pediatric endocrinologists, children with skeletal dysplasias have not been consistently managed by these physicians. Growth-altering treatments have broadened the role of the pediatric endocrinologist to…
Achondroplasia growth charts included in this article. Achondroplasia is the most common genetic skeletal disorder causing disproportionate short stature/dwarfism. Common additionalfeatures include spinal stenosis, midface retrusion, macrocephaly and a generalized…
These treatment pathways were developed by the type 2 diabetes subgroup of the PES Diabetes SIG, using evidence base when available in combination with expert clinical experience. They consist of…