Endocrine Abstracts

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...

Introduction: OPKO Biologics has produced a long-acting human growth hormone (hGH), MOD-4023, containing three copies of a naturally-occurring peptide (C-terminal peptide, CTP) that markedly increases growth hormone’s in vivo residence. We describe the development and validation of a pharmacokinetic (PK) and pharmacodynamic (PD) model to characterize the relationship between MOD-4023 dose, serum concentrations (Cserum), and IGF-1 responses in healthy adults, GHD adults, a...

Goal: To carry out a retrospective analysis of case histories of children and adolescents with genetically determined types of short stature who admitted a paediatric department of a clinic of Research Institute of Endocrinologyduring 2003–2013.Materials and methods: An analysis of case histories of children and adolescents with short stature 3 to 17 years who undertook inpatient treatment in paediatric department of RIE clinic during 2003–2013...

Introduction: Wolfram Syndrome is a rare autosomal recessive progressive neurodegenerative disorder with estimated prevalence of 1 in 500,000 r, also known as DIDMOAD syndrome for its four most common features (Diabetes Insipidus, Diabetes Mellitus, Optic Atrophy and Deafness). Patients present with nonautoimmune and non-HLA linked diabetes mellitus associated with optic atrophy in the first decade, diabetes insipidus and sensorineural deafness in the second decade, renal trac...

Objective: To investigate the changes in serum creatine kinase (CK) levels during the treatment of patients with growth hormone (GH).Methods: We evaluated 715 cases who were treated with GH retrospectively. The clinical data of 20 children with severe CK elevation (>500 IU/L) were analyzed.Results: CK elevation was recorded in 96 of the cases (13.5%) during GH theraphy. 20 of these patients had severe CK elevation. The mean age...

Aims: Attention Deficit Disorder & Hyperactivity Syndrome (ADHD) is identify attention deficit,impulsivity hyperactivity before the age of seven years old. ADHD is a serious disease which could cause corruption of daily lifetime and needed to be taken psychostimulant drugs. This case brings on that these kind of drugs can have unexplored side effects. In this study; it was reported 7 precocious puberty (PP) cases which used methylphenidate due to ADHD...

Introduction: T1DM is caused by immune mediated destruction of pancreatic β-cells, leading to partial, or absolute insulin deficiency.The most frequent age of onset is 12 years. IGF-1 is a polypeptide with 70 amino acids and its chemical structure is similar to insulin. IGF-1 is associated with early development and has anabolic effect in adults. IGF-1 regulates many physiological functions, including glucose metabolism, cell survival and proliferation.<p class="abste...

Neurofibromatosis 1 (NF1) is a rare disease determined by mutations in the RAS-MAPK pathway. It can cause precocious or delayed puberty.Case 1: A 11 years 9 months old girl known with Neurofibromatosis – Noonan Syndrome (NF-NS) was admitted for severe growth deficit (-5.14 SDS). She had over 20 café au lait spots, hypertelorism, pterigium colli, B1 P1. At 18 months she had had surgery for pulmonary stenosis and after that a left ventricular tum...