Burosumab well tolerated for XLH in infants younger than 1 year
Burosumab treatment for X-linked hypophosphataemia may begin in the first year of life, suggest phase 2 trial findings published in The Lancet Diabetes & Endocrinology.
Vosoritide may offer ‘precision medicine’ for MAPK pathway growth disorders
Basket trial findings indicate that vosoritide therapy may improve growth in children with short stature related to rare genetic disorders of the Ras–mitogen-active protein kinase pathway.
GnRHa therapy every 24 weeks comparable to 12-weekly formulation for CPP
UK study findings indicate that a slow-release formulation of the gonadotropin-releasing hormone analogue triporelin given every 24 weeks is as effective as the standard 12-weekly treatment for central precocious puberty.
Achondroplasia guidelines outline UK multidisciplinary care throughout childhood
The UK Achondroplasia Network has developed consensus guidelines on multidisciplinary care of children and young people with achondroplasia from diagnosis until adulthood.
SGA impaired glucose metabolism exacerbated by growth hormone therapy
Glucose–insulin impairment in small-for-gestational-age children is comparable to that found in children with obesity and worsened by use of growth hormone therapy, say German researchers.
European guidance released for childhood adrenal, extra-adrenal paraganglioma care
A European Standard Clinical Practice Guidance has been published in the European Journal of Endocrinology on the diagnosis and treatment of adrenal and extra-adrenal paragangliomas in children and adolescents.
Navepegritide APPROACH improves height velocity for children with achondroplasia
Treatment with a C-type natriuretic peptide prodrug significantly increases annualised growth velocity in children with achondroplasia and offers additional health benefits, say the APPROACH trial investigators.
CAH-specific growth charts aid clinical management during childhood
US researchers have created age- and sex-adjusted growth charts for use in children with classic congenital adrenal hyperplasia, reflecting the distinct patterns of height and weight among patients versus the general population.
Noonan syndrome consensus statements guide lifelong care
Italian clinicians have published a set of consensus statements on the multidisciplinary diagnosis, care and treatment of patients with Noonan syndrome from childhood and beyond.
Age at childhood differentiated thyroid cancer diagnosis linked to disease severity
Earlier age at diagnosis of childhood differentiated thyroid cancer is associated with a greater risk of advanced disease, but overall, patients have excellent long-term survival irrespective of age, stage, and treatment response, US study findings indicate.
