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Once-Daily, Subcutaneous Vosoritide Therapy in Children With Achondroplasia: A Randomised, Double-Blind, Phase 3, Placebo-Controlled, Multicentre Trial

Lancet, The, 2020
Paul Harmatz, Klane K White
exaly  

Quality of Life in Adults With Achondroplasia in the United States

American Journal of Medical Genetics, Part A, 2021
Elizabeth A Yonko, Cathleen L Raggio, Erin M Carter   +2 more
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Novel Therapeutic Approaches for the Treatment of Achondroplasia

Bone, 2020
Ravi Savarirayan, Laurence Legeai-Mallet
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Lifetime Impact of Achondroplasia: Current Evidence and Perspectives on the Natural History

Bone, 2021
Klaus Mohnike, Renée Shediac, Amaka C. Offiah   +2 more
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Growth Parameters in Children With Achondroplasia: A 7-Year, Prospective, Multinational, Observational Study

Genetics in Medicine, 2022
Paul Harmatz, Genevieve Baujat, Lynda E Polgreen   +2 more
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Birth Prevalence of Achondroplasia: A Systematic Literature Review and Meta-Analysis

American Journal of Medical Genetics, Part A, 2020
Renée Shediac, Sarah Landis, Pamela K Foreman   +2 more
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The Molecular and Genetic Basis of Fibroblast Growth Factor Receptor 3 Disorders: The Achondroplasia Family of Skeletal Dysplasias, Muenke Craniosynostosis, and Crouzon Syndrome With Acanthosis Nigricans

Endocrine Reviews, 2000
Clair A. Francomano, Zoltan Vajo
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