Results 131 to 140 of about 534 (161)
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Efficacy of vosoritide in the treatment of achondroplasia
Drugs of Today, 2022Achondroplasia is the commonest form of dwarfism and results from a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene on chromosome 4p16.3. The mutation is at nucleotide 1138 resulting in a G-to-A transition (134934.0001). This condition is characterized by full penetration meaning that everyone with this genetic mutation will exhibit ...
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BoneAchondroplasia (ACH), a skeletal dysplasia, is characterized by disproportionate short stature and impaired quality of life. In Japan, growth hormone (GH) therapy has been available since 1997, and vosoritide, a C-type natriuretic peptide analog, was approved in 2022.
Takuo Kubota +2 more
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Genetics in MedicinePurpose: Evaluate the impact of vosoritide on health-related quality of life in children with achondroplasia. Methods: Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled ...
Paul Harmatz +2 more
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Vosoritide, a Miracle Drug, Covering Unmet Need in Achondroplasia: A Regulatory Update
Intractable and Rare Diseases Research, 2023 Dwarfism is a rare condition characterized by small stature. Achondroplasia is predominantly considered the leading cause of dwarfism. Although the condition is not life-threatening, it dramatically impacts the social life of the patient. The United States Food and Drug Administration (US FDA) first approved the drug Voxzogo (vosoritide) for ...
Simran +4 more
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Real-World Safety and Effectiveness of Vosoritide in Achondroplasia: Results From a Single Center in Portugal [PDF]
Advances in TherapyChristoph Beger +2 more
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Effect of Vosoritide Therapy on IGF-I and Endogenous C-Type Natriuretic Peptide in Hypochondroplasia
Journal of Clinical Endocrinology and MetabolismCONTEXT: Insulin-like growth factor-1 (IGF-1) and C-type natriuretic peptide (CNP) promote endochondral bone growth. We previously reported that vosoritide, a CNP analog, increases annualized growth velocity (AGV) and height standard deviation (SD) in ...
Roopa Kanakatti Shankar +2 more
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Efficacy of vosoritide treatment in patients with achondroplasia
Journal of Pediatric Endocrinology and DiabetesObjectives: The objective was to evaluate the efficacy and safety profile of vosoritide treatment in children with achondroplasia after 1 year of administration. Material and Methods: This was a retrospective study including 14 ...
Pilar Morillas-Amat +5 more
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Role of Vosoritide Drug on Children's With Achondroplasia
World Journal of Current Medical and Pharmaceutical ResearchAchondroplasia is a genetic condition with an autosomal dominant inheritance pattern. It is caused by detrimental heterozygous FGFR3 gene mutations. Gain-of-function mutations in the FGFR3 gene impair chondrocyte differentiation and proliferation, which has a negative impact on the formation of new bone.
Ahmed tawfeeq Ahmed +2 more
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A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia
NEJM EvidenceHypochondroplasia, a fibroblast growth factor receptor 3 (FGFR3)-related skeletal condition characterized by disproportionate short stature and a spectrum of clinical features, has no available targeted therapies. Vosoritide, a C-type natriuretic peptide analogue approved for the treatment of achondroplasia, is being investigated for hypochondroplasia ...
Andrew, Dauber +29 more
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Potential Efficacy of Vosoritide for Foramen Magnum Stenosis in a Patient With Achondroplasia
Pediatrics InternationalNaoto Nishizaki +2 more
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