Anastrozole may aid height growth for children with 21OHD CAH
Some children with congenital adrenal hyperplasia (CAH) could benefit from treatment with the aromatase inhibitor anastrozole to optimise their growth potential, US researchers suggest.
Encouraging oral infigratinib safety and efficacy profile reported for achondroplasia
Once daily oral treatment with infigratinib has a tolerable side effect profile and a significant positive benefit on both annualised height velocity and upper-to-lower body segment ratio in children with achondroplasia, indicates research published in The New England Journal of Medicine.
Minipuberty hormonal changes characterised for infant girls
A study of infant girls has characterised the hormonal changes of minipuberty and uncovered how the ovaries, uterus and reproductive hormones interrelate during this period.
Novel Silver–Russell syndrome genetic variants help unravel genotype–phenotype relationships
Researchers have identified novel pathogenic variants that may account for approximately 9% of children with Silver–Russell syndrome who have not previously had a molecular diagnosis and aid understanding of the relationships between genotype and phenotype.
Liraglutide reduces BMI in prepubertal children with severe obesity
Use of the glucagon-like peptide -1 receptor agonist liraglutide alongside diet and exercise guidance significantly reduces BMI in children aged 6 to 11 years with severe obesity compared with lifestyle interventions alone, shows the SCALE Kids Trial.
REAL5 update of somapacitan continues to support weekly hGH therapy for SGA children
One-year findings from the REAL5 trial show a continued growth response with use of long-acting human growth hormone treatment with somapacitan at a dose of 0.24 mg/kg per week in children born small for gestational age.
Phase 3 crinecerfont therapy findings may expand medication options for classical CAH
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 may be avoided for some pediatric papillary thyroid carcinoma patients
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 frequently missed by newborn screening
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.
Congenital hyperinsulinism phenotypes, genotypes linked to response to therapy
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.