ESPE Yearbook of Paediatric Endocrinology

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

Published by BioScientifica
Previous issue | Volume 18 | ESPEYB18 | Next issue

Yearbook of Paediatric Endocrinology 2021

5. Bone, Growth Plate and Mineral Metabolism

Novel treatments for rare skeletal disorders

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Marta Baroncelli, Adalbert Raimann, Raja Padidela, Ola Nilsson
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Advances in clinical practice

ey0018.5-1 | Novel treatments for rare skeletal disorders | ESPEYB18

5.1. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial

R Savarirayan , L Tofts , M Irving , W Wilcox , CA Bacino , J Hoover-Fong , R Ullot Font , P Harmatz , F Rutsch , MB Bober , LE Polgreen , I Ginebreda , K Mohnike , J Charrow , D Hoernschemeyer , K Ozono , Y Alanay , P Arundel , S Kagami , N Yasui , KK White , HM Saal , A Leiva-Gea , F Luna-Gonzalez , H Mochizuki , D Basel , DM Porco , K Jayaram , E Fisheleva , A Huntsman-Labed , J Day

Lancet. 2020 Sep 5;396(10252):684–692. Abstract: https://pubmed.ncbi.nlm.nih.gov/32891212/In brief: Activating mutations in FGFR3 inhibit endochondral ossification in achondroplasia resulting in disproportionate extreme short stature. In this randomised, double-blind, phase 3, placebo-controlled trial, once-daily subcutaneous treatment with vosoritide, a C-type natriuretic pep...
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ey0018.5-2 | Novel treatments for rare skeletal disorders | ESPEYB18

5.2. Evaluation of FGFR inhibitor ASP5878 as a drug candidate for achondroplasia

Ozaki Tomonori , Kawamoto Tatsuya , Iimori Yuki , Takeshita Nobuaki , Yamagishi Yukiko , Nakamura Hiroaki , Kamohara Masazumi , Fujita Kaori , Tanahashi Masayuki , Tsumaki Noriyuki

Sci Rep. 2020 Dec 1;10(1):20915 Abstract: https://pubmed.ncbi.nlm.nih.gov/33262386/In brief: Inhibition of excessive FGFR3 signalling constitutes the key mechanism of pharmacological treatments in achondroplasia. In this pharmacokinetic and pharmacodynamic animal study, the FGFR inhibitor ASP5878 with potential oral application mode revealed beneficial effects on skeletal growth in...
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ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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