ISSN 1662-4009 (online)

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Yearbook of Paediatric Endocrinology 2019

5. Bone, Growth Plate and Mineral Metabolism

New Therapies and Novel Therapeutic Strategies

ey0016.5-1 | New Therapies and Novel Therapeutic Strategies | ESPEYB16

5.1. Cartilage-targeted IGF-1 treatment to promote longitudinal bone growth

JC Lui , M Colbert , CSF Cheung , M Ad , A Lee , Z Zhu , KM Barnes , DS Dimitrov , J Baron

Abstract: Mol. Ther. 2019;27:673–680.In brief: The authors developed a fusion protein containing a cartilage-targeting antibody fragment and Insulin-like growth factor 1 (Igf1) and demonstrate that it can stimulate growth plate cartilage at lower and less frequent doses than Igf1. This is a novel approach that paves the way for the development of tissue-specific target...

ey0016.5-2 | New Therapies and Novel Therapeutic Strategies | ESPEYB16

5.2. Humanin is a novel regulator of Hedgehog signaling and prevents glucocorticoid-induced bone growth impairment

F Zaman , Y Zhao , B Celvin , HH Mehta , J Wan , D Chrysis , C Ohlsson , B Fadeel , P Cohen , L Savendahl

Abstract: FASEB J. 2019;33:4962–4974.In brief: Glucocorticoid-induced growth impairment is reverted by the mitochondrial peptide humanin in dexamethasone-treated mice without attenuation of anti-inflammatory effects.Comment: Humanin (HN) is a 24 aminoacid peptide that was originally discovered as a neuroprotective factor and later shown ...

ey0016.5-3 | New Therapies and Novel Therapeutic Strategies | ESPEYB16

5.3. ENPP1 enzyme replacement therapy improves blood pressure and cardiovascular function in a mouse model of generalized arterial calcification of infancy

T Khan , KW Sinkevicius , S Vong , A Avakian , MC Leavitt , H Malanson , A Marozsan , KL Askew

Abstract: Dis Model Mech. 2018 Oct 1; 11(10): dmm035691.In brief: Generalized arterial calcification of infancy (GACI) is a severe, rare disease characterized by excessive calcification of large and medium sized arteries caused by homozygous loss-of-function mutations in ENPP1. Here, in Enpp1 deficient mice, treatment with recombinant hu...