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ey0016.2-4 | Neonatal Hypoglycaemia | ESPEYB16

2.4. Sirolimus: efficacy and complications in children with hyperinsulinemic hypoglycemia: A 5-year follow-up study

G Maria , D Antonia , A Michael , M Kate , E Sian , FE Sarah , D Mehul , S Pratik

To read the full abstract: J Endocr Soc. 2019 Feb 7;3(4):699713.This paper describes a retrospective study of patients with congenital hyperinsulinism (CHI) who were treated with mammalian target of rapamycin (mTOR) inhibitor, sirolimus, in a tertiary service, and reviews the 15 publications reporting CHI patients treated with sirolimus.The diffuse forms of C...

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ey0020.6-6 | Clinical Trials – New Treatments | ESPEYB20

6.6. Crinecerfont, a CRF1 receptor antagonist, lowers adrenal androgens in adolescents with congenital adrenal hyperplasia

Newfield Ron S , Sarafoglou Kyriakie , Fechner Patricia Y , Nokoff Natalie J , Auchus Richard J , Vogiatzi Maria G , Jeha George S , Giri Nagdeep , Roberts Eiry , Sturgeon Julia , Chan Jean L , Farber Robert H

Brief summary: This study evaluated the safety, tolerability and efficacy of Crinecerfont, a CRF1R antagonist in adolescents with classic congenital adrenal hyperplasia (CAH).Classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21OHD) is characterized by impaired cortisol synthesis and excess adrenal androgen secretion. Sufficient suppression of adrenal andogen production in classic CAH may be difficult with current formulations...

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ESPE Yearbook of Paediatric Endocrinology

2.4. Sirolimus: efficacy and complications in children with hyperinsulinemic hypoglycemia: A 5-year follow-up study

6.6. Crinecerfont, a CRF1 receptor antagonist, lowers adrenal androgens in adolescents with congenital adrenal hyperplasia

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