Novel Treatments for Rare Skeletal Disorders

ESPE Yearbook of Paediatric Endocrinology

ISSN 1662-4009 (online)

An indispensable reference tool endorsed by the European Society for Paediatric Endocrinology

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Yearbook of Paediatric Endocrinology 2023

3. Bone, Growth Plate and Mineral Metabolism

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Raja Padidela, Ameya Bendre, Lars Ottosson, Ola Nilsson

Table of contents

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Advances in Clinical Practice

ey0020.3-1 | Novel Treatments for Rare Skeletal Disorders

3.1. Safety and efficacy of denosumab for fibrous dysplasia of bone

ey0020.3-2 | Novel Treatments for Rare Skeletal Disorders

3.2. Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long-acting C-type natriuretic peptide prodrug, TransCon CNP

ey0020.3-3 | Novel Treatments for Rare Skeletal Disorders

3.3. Burosumab treatment for autosomal recessive hypophosphatemic rickets type 1 (ARHR1)

ESPE Yearbook of Paediatric Endocrinology
ISSN 1662-4009 (Online)
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