Noonan syndrome growth not improved with simvastatin therapy
Simvastatin treatment has not significantly improved growth in a randomised phase 3 trial of children with Noonan syndrome (NS), French researchers report in eClinicalMedicine.
Phenotype expanded for children with Temple syndrome
Temple syndrome (TS14) has a broader range of phenotypic features than previously recognised and the presentation may change with increasing age, indicates an analysis of children attending two European referral centres.
Setmelanotide combats hypothalalmic obesity in children and adults
The TRANSECEND trial confirms that the melanocortin-4 receptor agonist setmelanotide achieves a significantly greater reduction in BMI and hunger than placebo in children, adolescents and adults with acquired hypothalamic obesity.
Guideline released for central precocious puberty diagnosis and treatment
The Endocrine Society has published a clinical practice guideline on the diagnosis and treatment of children with central precocious puberty in The Journal of Clinical Endocrinology & Metabolism.
New paediatric endocrine guidance for transition and short stature genetic testing
International guidance has been published on managing the transition of children with endocrine disorders from paediatric to adult services, and on the use of genetic testing in children with short stature.
Early-stage type 1 diabetes screening ‘efficient’ in general childhood population
Islet autoantibody screening for early-stage type 1 diabetes in children is feasible in the general population, the Fr1da study investigators have found.
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.
