The first global phase 2 study of vosoritide showed an average increased growth rate of 1.8 cm per year in children with hypochondroplasia, a genetic cause of short stature in children, according to a paper recently published in eClinicalMedicine. The clinical trial is funded by BioMarin. Researchers led by Andrew Dauber, MD, chief of Endocrinology at Children’s...
On the 20th anniversary of the U.S. Food and Drug Administration’s approval of growth hormone (GH) treatment for idiopathic short stature (ISS), Adda Grimberg, MD, and Colin P. Hawkes, MD, PhD, both of the Children’s Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania, published a mini-review in The Journal...
Childhood cancer patients and survivors are at increased risk for growth disturbances, for myriad reasons, and it is important to periodically monitor their growth, according to a paper recently published in The Journal of Clinical Endocrinology & Metabolism. Tomoko Yoshida, MD, PhD, and Angela Delaney, MD, both of St. Jude Children’s Research Hospital in Memphis,...
The increase in free insulin-like growth factor (IGF)-I levels in prepubertal children with Prader-Willi Syndrome (PWS) treated with exogenous growth hormone (GH) could be caused by increased pregnancy-associated plasma protein A (PAPP-A, PAPP-A2) levels and a reduction in stanniocalcins (STC-1, STC-2), according to a study recently published in The Journal of Clinical Endocrinology & Metabolism....
Lonapegsomatropin, a once-weekly prodrug of somatropin, can help children in Tanner Stages 3-5 maintain consistent height standard deviation scores (SDS) without an increase in the mean dose, according to a study presented at ENDO 2023. Ascendis Pharma is marketing the drug as TransCon hGH. Researchers led by Paul Hofman, MD, FRACP, of the Liggins Institute...
Children with achondroplasia (ACH), the most common form of hereditary short stature, experienced statistically significant improvements in their annual height velocity with the prodrug TransCon CNP, according to a study presented at ENDO 2023. Ascendis Pharma is investigating the drug in the Phase 2 ACcomplisH trial. Researchers led by Ravi Savarirayan, MBBS, FRACP, MD, of...
In otherwise healthy short children, quality of life and self-esteem are associated with coping skills and how supported they feel, not the degree of their short stature, according to a study recently published in The Journal of Pediatrics. Researchers led by Adda Grimberg, MD, a pediatric endocrinologist and Scientific Director of the Growth Center at Children’s...
Pediatric growth hormone deficiency treatments could soon be painless. Fernando Cassorla, MD, is traveling from Chile to Chicago to present research at ENDO 2023 that could potentially change the way pediatric hormone treatments are administered. If a pill could replace painful injections, patients and caregivers alike can finally wipe away their tears. A convenient, well...
Pediatric growth hormone deficiency treatments could soon be painless. Fernando Cassorla, MD, is traveling from Chile to Chicago to present research at ENDO 2023 that could potentially change the way pediatric hormone treatments are administered. If a pill could replace painful injections, patients and caregivers alike can finally wipe away their tears. A convenient, well...
