Endocrine Abstracts

Mammalian growth is an extraordinarily complex phenomenon, regulated by a panoply of genetic and environmental factors. In light of this, it is all the more extraordinary that the combination of targeted gene knock-out studies and human mutational analysis has convincingly demonstrated that the Insulin-like growth factor (IGF) system is responsible for a significant portion of both intrauterine and postnatal growth. IGF production in utero is essentially growth hormone ...

Ron G Rosenfeld, Lucile Packard Foundation for Children's Health, Stanford University, Palo Alto, CA., USA. AbstractRon G Rosenfeld, MD, is the Senior Vice-President for Medical Affairs at the Lucile Packard Foundation for Children's Health at Stanford University. He is Professor of Pediatrics at Stanford University, as well as Professor and Chair (emeritus) of Pediatrics and of Cell and Developmental Biology at Oregon...

Patients originally diagnosed with IsGHD may develop additional pituitary hormone deficiencies later in life. This study aimed to identify factors that predict development of MPHD and to characterize the time course of specific hormone deficiencies. Pediatric patients with IsGHD were from an observational study (GeNeSIS). Additional hormone deficiency during follow-up was accepted, if indicated by check box on the case report forms, by an adverse event or by starting replaceme...

Objective: Growth hormone (GH) replacement therapy currently requires daily injections. This may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) has been developed for once-weekly administration in growth hormone deficient (GHD) adults and children. The 18 and 24 month efficacy of once-weekly subcutaneous (SC) administration of MOD-4023 was evaluated in a Phase 2 study in children with GH deficiency.Design and ...

Objective: Growth hormone (GH) replacement therapy currently requires daily injections. This may cause poor compliance, inconvenience and distress for patients. CTP-modified human GH (MOD-4023) has been developed for once-weekly administration in growth hormone-deficient (GHD) adults and children. In the present Phase 2 study, the safety and tolerability of once-weekly subcutaneous (SC) administration of MOD-4023 were evaluated in GHD children.Design and...

Background: Acid-labile subunit (ALS) deficiency inhibits ternary complex formation leading to primary IGF1 deficiency and short stature. Potential metabolic consequences such as diabetes and low bone mass are not well studied.Objective: This study measured insulin sensitivity, lipid profile, bone density and structure in members of 4 affected families and explore possible gene-dose effects.Methods: Four patients (7–21 years) ...