Ron G. Rosenfeld
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View article: IGF-1 Assessment During Weekly Somatrogon Treatment in Pediatric Patients With GH Deficiency
IGF-1 Assessment During Weekly Somatrogon Treatment in Pediatric Patients With GH Deficiency Open
Context In patients with GH deficiency (GHD) receiving GH treatment, IGF-1 concentrations are used by physicians to monitor treatment safety and efficacy and guide dosing decisions. Somatrogon is a long-acting GH approved as a once-weekly …
View article: Role of the GH-IGF Axis in Statural Growth and Harmonious Body Proportionality: In Search of Vitruvian Man?
Role of the GH-IGF Axis in Statural Growth and Harmonious Body Proportionality: In Search of Vitruvian Man? Open
Context Body proportions are the objective parameters of harmonious growth and reflect the interplay of genetic, environmental, metabolic, and hormonal actions. Mutations in the growth hormone receptor gene (GHR) result in severe growth fa…
View article: What Is the Role for Pediatric Endocrinologists in the Management of Skeletal Dysplasias?
What Is the Role for Pediatric Endocrinologists in the Management of Skeletal Dysplasias? Open
Children with skeletal dysplasias have not been consistently managed by pediatric endocrinologists despite the recognized expertise of these practitioners in managing genetic growth disorders. Growth-altering treatments have broadened the …
View article: THU170 Clinical And Immunological Response To Somatrogon In Two Siblings With A Homozygous Whole Gene Deletion Of The Growth Hormone 1 Gene
THU170 Clinical And Immunological Response To Somatrogon In Two Siblings With A Homozygous Whole Gene Deletion Of The Growth Hormone 1 Gene Open
Disclosure: R. Gupta: None. R. Khadgawat: None. R.G. Rosenfeld: Advisory Board Member; Self; Lumos, DNARx, BioMarin. Consulting Fee; Self; OPKO Health. M.T. Dattani: Advisory Board Member; Self; Ferring Pharmaceuticals, Novo Nordisk, Pfize…
View article: OR21-04 Long-term Efficacy And Safety Of Once-weekly Somatrogon In Pediatric Subjects With Growth Hormone Deficiency: Results From Up To 8 Years Of Somatrogon Treatment
OR21-04 Long-term Efficacy And Safety Of Once-weekly Somatrogon In Pediatric Subjects With Growth Hormone Deficiency: Results From Up To 8 Years Of Somatrogon Treatment Open
Disclosure: Z. Zadik: None. N. Zelinska: Consulting Fee; Self; Novo Nordisk, Berlin-Chemie, Medtronic, Sanofi-Aventis. Research Investigator; Self; MacroGenics, Novo Nordisk, Pfizer, Inc., Merck, OPKO Health, Ferring Pharmaceuticals, Teva …
View article: An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment
An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment Open
Objectives Somatrogon is a long-acting recombinant human growth hormone (GH) employed as a once-weekly treatment for children with GH deficiency (GHD). A 12-month, phase 2 study of once-weekly somatrogon vs. once-daily GH (Genotropin ® ) w…
View article: RF26 | PSAT150 Growth Outcomes From the Phase 2 and Phase 3 Studies of Once Weekly Somatrogon vs Daily Genotropin in Pediatric Patients With Growth Hormone Deficiency: Comparisons With Published Literature and an International Growth Study Database
RF26 | PSAT150 Growth Outcomes From the Phase 2 and Phase 3 Studies of Once Weekly Somatrogon vs Daily Genotropin in Pediatric Patients With Growth Hormone Deficiency: Comparisons With Published Literature and an International Growth Study Database Open
Background Somatrogon is a long-acting recombinant human growth hormone (hGH) currently in development as a once-weekly injectable treatment for children with growth hormone deficiency (GHD). In a phase 2 (NCT01592500) and a phase 3 (NCT02…
View article: Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study
Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study Open
Background Achondroplasia is associated with disproportionate short stature and significant and potentially severe medical complications. Vosoritide is the first medicine to treat the underlying cause of achondroplasia and data from phase …
View article: Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study
Efficacy and Safety of Once-Weekly Somatrogon Compared with Once-Daily Somatropin (Genotropin®) in Japanese Children with Pediatric Growth Hormone Deficiency: Results from a Randomized Phase 3 Study Open
Introduction: Somatrogon is a long-acting recombinant human growth hormone being developed as a once-weekly treatment for children with growth hormone deficiency (GHD). The objective of this phase 3 study (NCT03874013) was to…
View article: Pregnancy-Associated Plasma Protein (PAPP)-A2 in Physiology and Disease
Pregnancy-Associated Plasma Protein (PAPP)-A2 in Physiology and Disease Open
The growth hormone (GH)/insulin-like growth factor (IGF) axis plays fundamental roles during development, maturation, and aging. Members of this axis, composed of various ligands, receptors, and binding proteins, are regulated in a tissue-…
View article: Prolonged, Controllable Protein Production of cDNA-Encoded hGH Maintained at Therapeutic Serum Levels Following One Systemic Administration of a Non-Viral, Non-Integrating, DNA-and Liposome-Based <i>in vivo</i> Gene Therapy Platform in Immunocompetent Mice
Prolonged, Controllable Protein Production of cDNA-Encoded hGH Maintained at Therapeutic Serum Levels Following One Systemic Administration of a Non-Viral, Non-Integrating, DNA-and Liposome-Based <i>in vivo</i> Gene Therapy Platform in Immunocompetent Mice Open
Recombinant human growth hormone (rhGH), the mainstay of hGH replacement therapy, is injected daily for years to enable children to achieve normal stature. Daily rhGH injections are required because its serum-T1/2 is <20 minutes. Human …
View article: Phase 3 Study Evaluating Once Weekly Somatrogon Compared to Daily Genotropin in Japanese Patients With Pediatric Growth Hormone Deficiency (pGHD)
Phase 3 Study Evaluating Once Weekly Somatrogon Compared to Daily Genotropin in Japanese Patients With Pediatric Growth Hormone Deficiency (pGHD) Open
Objectives: Somatrogon is a long-acting recombinant human growth hormone consisting of the amino acid sequence of human growth hormone and three copies of the carboxy-terminal peptide of human chorionic gonadotropin. Somatrogon is being de…
View article: Results From an Open-Label Extension of the Phase 2 Dose Finding Study of Once Weekly Somatrogon vs Daily Genotropin in Pediatric Patients With Growth Hormone Deficiency (GHD)
Results From an Open-Label Extension of the Phase 2 Dose Finding Study of Once Weekly Somatrogon vs Daily Genotropin in Pediatric Patients With Growth Hormone Deficiency (GHD) Open
Background: Somatrogon, a long-acting recombinant human growth hormone, is being developed as a once weekly treatment for pediatric patients (pts) with GHD. A phase 2, 12 month study (NCT01592500) in pts with GHD showed that weekly somatro…
View article: GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults
GH safety workshop position paper: a critical appraisal of recombinant human GH therapy in children and adults Open
Recombinant human GH (rhGH) has been in use for 30 years, and over that time its safety and efficacy in children and adults has been subject to considerable scrutiny. In 2001, a statement from the GH Research Society (GRS) concluded that ‘…
View article: Genetic causes of growth hormone insensitivity beyond GHR
Genetic causes of growth hormone insensitivity beyond GHR Open
View article: Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations: Table 1
Growth Hormone Research Society perspective on the development of long-acting growth hormone preparations: Table 1 Open
The Growth Hormone (GH) Research Society (GRS) convened a workshop to address important issues regarding trial design, efficacy, and safety of long-acting growth hormone preparations (LAGH).
View article: Supplementary Material for: A Novel Mutation in Insulin-Like Growth Factor 1 Receptor (c.641-2A>G) Is Associated with Impaired Growth, Hypoglycemia, and Modified Immune Phenotypes
Supplementary Material for: A Novel Mutation in Insulin-Like Growth Factor 1 Receptor (c.641-2A>G) Is Associated with Impaired Growth, Hypoglycemia, and Modified Immune Phenotypes Open
Introduction: Insulin-like growth factor 1 receptor (IGF1R) mutations lead to systemic disturbances in growth and glucose homeostasis due to widespread IGF1R expression throughout the body. IGF1R is expressed by innate and ad…
View article: A Novel Mutation in Insulin-Like Growth Factor 1 Receptor (c.641-2A&#x3e;G) Is Associated with Impaired Growth, Hypoglycemia, and Modified Immune Phenotypes
A Novel Mutation in Insulin-Like Growth Factor 1 Receptor (c.641-2A>G) Is Associated with Impaired Growth, Hypoglycemia, and Modified Immune Phenotypes Open
Introduction: Insulin-like growth factor 1 receptor (IGF1R) mutations lead to systemic disturbances in growth and glucose homeostasis due to widespread IGF1R expression throughout the body. IGF1R is expressed by innate and ad…
View article: A Novel Homozygous Mutation of the Acid-Labile Subunit <i>(IGFALS)</i> Gene in a Male Adolescent
A Novel Homozygous Mutation of the Acid-Labile Subunit <i>(IGFALS)</i> Gene in a Male Adolescent Open
Acid-labile subunit (ALS) forms ternary complexes with insulin like growth factor-1 (IGF-1) and IGF-binding protein-3 (IGFBP-3) and is essential for normal circulating IGF-1 levels. The IGFALS gene encodes the ALS and mutations in <…
View article: Supplementary Material for: Height Gain and Safety Outcomes in Growth Hormone-Treated Children with Idiopathic Short Stature: Experience from a Prospective Observational Study
Supplementary Material for: Height Gain and Safety Outcomes in Growth Hormone-Treated Children with Idiopathic Short Stature: Experience from a Prospective Observational Study Open
Background/Objectives: Growth hormone (GH) treatment of idiopathic short stature (ISS) received US Food and Drug Administration approval in 2003. We assessed height gain and safety in 2,450 children with ISS treated with GH i…
View article: Diagnosis, Genetics, and Therapy of Short Stature in Children: A Growth Hormone Research Society International Perspective
Diagnosis, Genetics, and Therapy of Short Stature in Children: A Growth Hormone Research Society International Perspective Open
The Growth Hormone Research Society (GRS) convened a Workshop in March 2019 to evaluate the diagnosis and therapy of short stature in children. Forty-six international experts participated at the invitation of GRS including clinicians, bas…
View article: Nonclassical GH Insensitivity: Characterization of Mild Abnormalities of GH Action
Nonclassical GH Insensitivity: Characterization of Mild Abnormalities of GH Action Open
GH insensitivity (GHI) presents in childhood with growth failure and in its severe form is associated with extreme short stature and dysmorphic and metabolic abnormalities. In recent years, the clinical, biochemical, and genetic characteri…
View article: Screening a large pediatric cohort with GH deficiency for mutations in genes regulating pituitary development and GH secretion: Frequencies, phenotypes and growth outcomes
Screening a large pediatric cohort with GH deficiency for mutations in genes regulating pituitary development and GH secretion: Frequencies, phenotypes and growth outcomes Open
View article: Safety Outcomes During Pediatric GH Therapy: Final Results From the Prospective GeNeSIS Observational Program
Safety Outcomes During Pediatric GH Therapy: Final Results From the Prospective GeNeSIS Observational Program Open
Context Safety concerns have been raised regarding premature mortality, diabetes, neoplasia, and cerebrovascular disease in association with GH therapy. Objective To assess incidence of key safety outcomes. Design Prospective, multinationa…
View article: Dominant-negative STAT5B mutations cause growth hormone insensitivity with short stature and mild immune dysregulation
Dominant-negative STAT5B mutations cause growth hormone insensitivity with short stature and mild immune dysregulation Open
Growth hormone (GH) insensitivity syndrome (GHIS) is a rare clinical condition in which production of insulin-like growth factor 1 is blunted and, consequently, postnatal growth impaired. Autosomal-recessive mutations in signal transducer …
View article: Growth Hormone Research Society perspective on biomarkers of GH action in children and adults
Growth Hormone Research Society perspective on biomarkers of GH action in children and adults Open
Objective The Growth Hormone Research Society (GRS) convened a Workshop in 2017 to evaluate clinical endpoints, surrogate endpoints and biomarkers during GH treatment of children and adults and in patients with acromegaly. Participants GRS…
View article: Screening a Large Pediatric Cohort with GH Deficiency for Mutations in Genes Regulating Pituitary Development and GH Secretion: Frequencies, Phenotypes and Growth Outcomes
Screening a Large Pediatric Cohort with GH Deficiency for Mutations in Genes Regulating Pituitary Development and GH Secretion: Frequencies, Phenotypes and Growth Outcomes Open
View article: Mortality in Children Receiving Growth Hormone Treatment of Growth Disorders: Data From the Genetics and Neuroendocrinology of Short Stature International Study
Mortality in Children Receiving Growth Hormone Treatment of Growth Disorders: Data From the Genetics and Neuroendocrinology of Short Stature International Study Open
Context Although pediatric growth hormone (GH) treatment is generally considered safe for approved indications, concerns have been raised regarding potential for increased risk of mortality in adults treated with GH during childhood. Objec…
View article: Biology of the somatotroph axis (after the pituitary)
Biology of the somatotroph axis (after the pituitary) Open
View article: Novel Dominant-Negative GH Receptor Mutations Expands the Spectrum of GHI and IGF-I Deficiency
Novel Dominant-Negative GH Receptor Mutations Expands the Spectrum of GHI and IGF-I Deficiency Open
Context: Autosomal-recessive mutations in the growth hormone receptor (GHR) are the most common causes for primary growth hormone insensitivity (GHI) syndrome with classical GHI phenotypically characterized by severe short stature and mark…