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Phase 3 trial findings support the use of an oral corticotropin-releasing factor type 1 receptor antagonist for the treatment of classic congenital adrenal hyperplasia in children with 21-hydroxylase deficiency.
Central congenital hypothyroidism is frequently missed in Israeli infants despite a newborn screening programme for thyroid dysfunction, shows research published in the Journal of Clinical Endocrinology & Metabolism.
Italian researchers have determined a glucagon stimulation test threshold that can predict whether a teenager with growth hormone deficiency has or has not experienced normalisation of GH levels at the time of transition to adult care.

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Phase 3 trial findings support the use of an oral corticotropin-releasing factor type 1 receptor antagonist for the treatment of classic congenital adrenal hyperplasia in children with 21-hydroxylase deficiency.
Radioactive iodine therapy may be avoided for some children undergoing surgery for papillary thyroid carcinoma, suggest findings from a study that shows good outcomes even for those with cervical lymph node metastases.
Central congenital hypothyroidism is frequently missed in Israeli infants despite a newborn screening programme for thyroid dysfunction, shows research published in the Journal of Clinical Endocrinology & Metabolism.
Researchers have characterised the pathogenesis, prognosis, treatment, neurological sequelae and prevalence of congenital hyperinsulinism in children born in Norway, finding genetic alterations to ABCC8 to be the most common cause of the condition.
Italian researchers have determined a glucagon stimulation test threshold that can predict whether a teenager with growth hormone deficiency has or has not experienced normalisation of GH levels at the time of transition to adult care.
Dutch research indicates that health-related quality of life remains largely stable over the 12 years after completion of growth hormone therapy for short stature in children who were born small for gestational age.
Genetic testing can lead to a diagnosis in around three quarters of children with short stature from consanguineous families, suggests a study of Turkish patients.
A whole-exome sequencing study has identified novel genes involved in the development of spontaneous congenital hypopituitarism in a large group of unrelated children from Argentina.
Phase 2 trial results support use of the melanocortin-4 receptor agonist setmelanotide for the treatment of rapid excess weight gain and hunger in patients with hypothalamic obesity.
Vosoritide treatment appears to increase growth velocity in children with hypochondroplasia and is well tolerated, show study findings
Most transgender adolescents treated with a gonadotropin-releasing hormone (GnRH) agonist plus oestradiol or testosterone reach an adult height that is within 2 standard deviations of that expected for their sex assigned at birth, research shows.
The majority of sporadic medullary thyroid cancer cases in children may be positive for RET mutations that are potentially amenable to treatment with a targeted agent, suggests research published in the Journal of Clinical Endocrinology & Metabolism.
Martin Savage
Programme Director
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