Results 141 to 150 of about 534 (161)
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Canadian Journal of Health Technologies
Canada’s Drug Agency (CDA-AMC) recommends that Voxzogo not be reimbursed by public drug plans for increasing linear growth in patients with achondroplasia (ACH) who are 4 months of age and older whose epiphyses are not closed. The Canadian Drug Expert Committee (CDEC) concluded that it is uncertain whether Voxzogo demonstrates acceptable clinical ...
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Canada’s Drug Agency (CDA-AMC) recommends that Voxzogo not be reimbursed by public drug plans for increasing linear growth in patients with achondroplasia (ACH) who are 4 months of age and older whose epiphyses are not closed. The Canadian Drug Expert Committee (CDEC) concluded that it is uncertain whether Voxzogo demonstrates acceptable clinical ...
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Early Neonatal Administration of Vosoritide in Achondroplasia: A Report of Two Cases
American Journal of Medical Genetics Part AABSTRACT Vosoritide, a C‐type natriuretic peptide analogue that inhibits FGFR3 signaling, is approved for the treatment of achondroplasia (ACH) from birth in Japan, Australia, and the United States; however, data on neonatal use remain limited.
Shinichiro Sano +5 more
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European Journal of Pediatrics
Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene. Vosoritide activates the NPR-B receptor to inhibit the overactive FGFR3 signaling pathway. We aim to pool the efficacy and safety outcomes of vosoritide in children with genetically confirmed achondroplasia who are receiving the approved dose of 15 μg/kg/day.
Ghaidaa A. Alfaraj +3 more
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Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene. Vosoritide activates the NPR-B receptor to inhibit the overactive FGFR3 signaling pathway. We aim to pool the efficacy and safety outcomes of vosoritide in children with genetically confirmed achondroplasia who are receiving the approved dose of 15 μg/kg/day.
Ghaidaa A. Alfaraj +3 more
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A randomized controlled trial of vosoritide in infants and toddlers with Achondroplasia
Zeitschrift für Geburtshilfe und Neonatologie, 2023L. E. Polgreen +21 more
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Vosoritide for Dominantly Inherited Short Stature
Case Medical Research, 2020openaire +1 more source
OP021: Vosoritide for children with achondroplasia: Growth velocity and pubertal milestones
Genetics in Medicine, 2022Melita Irving +17 more
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Use of vosoritide in children with achondroplasia - clinical experience
Endocrine AbstractsAhmet Kahveci +6 more
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Vosoritide in the treatment of achondroplasia
Revista Española Endocrinología Pediátricaopenaire +1 more source