Results 81 to 90 of about 1,955 (223)
Clinical and Translational Science, Volume 18, Issue 1, January 2025.ABSTRACT Monoclonal antibodies (mAbs) are critical components in the therapeutic landscape, but their dosing strategies often evolve post‐approval as new data emerge. This review evaluates post‐marketing label changes in dosing information for FDA‐approved mAbs from January 2015 to September 2024, with a focus on both initial and extended indications ...
Nai Lee +6 more
wiley +1 more source
Frontiers in Endocrinology, 2021 X-linked hypophosphatemic rickets (XLH) is the commonest inherited form of rickets. It is caused by an impaired regulation of fibroblast growth factor 23 (FGF23) due to a PHEX gene mutation, which leads to reduced tubular reabsorption of phosphate and ...
Giampiero Igli Baroncelli, Stefano Mora
doaj +1 more source
The international X-linked hypophosphataemia (XLH) registry (NCT03193476): rationale for and description of an international, observational study [PDF]
, 2020 Background X-linked hypophosphataemia (XLH) is a rare, hereditary, progressive and lifelong phosphate wasting disorder characterised by pathological elevations in fibroblast growth factor (FGF) 23 concentration and activity; XLH has an incidence of ...
Ariceta, Gema +11 more
core +6 more sources
Clinical Pharmacology &Therapeutics, Volume 116, Issue 6, Page 1560-1571, December 2024.The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) oversee pharmaceutical regulations, including orphan drugs targeting rare diseases with limited patient populations. Post‐marketing studies are crucial for monitoring safety and efficacy, with post‐marketing requirements (PMRs) mandated by the regulatory agencies to ...
Jae Hyeon Yu +5 more
wiley +1 more source
Tertiary hyperparathyroidism in two paediatric patients with X-linked hypophosphatemia during Burosumab treatment [PDF]
Introduction: Although secondary hyperparathyroidism is known in X-linked hypophosphatemia (XLH) patients receiving treatment, tertiary hyperparathyroidism with hypercalcemia is rare, especially in children.
Boot, Annemieke M. +2 more
core +1 more source
OR13-1 Burosumab Improves the Biochemical, Skeletal, and Clinical Symptoms of Tumor-Induced Osteomalacia Syndrome [PDF]
, 2019 Tumor-induced Osteomalacia (TIO) and Epidermal Nevus Syndrome with osteomalacia (ENS) are rare conditions in which ectopic production of FGF23 by tumor (TIO) and bone (ENS) lead to renal phosphate wasting, impaired 1,25(OH)2D synthesis, osteomalacia ...
Carpenter, Thomas +10 more
core +1 more source
Clinical Pharmacology &Therapeutics, Volume 116, Issue 5, Page 1343-1351, November 2024.Burosumab is indicated for treatment of a rare bone disease, X‐linked hypophosphatemia (XLH). The aim of this analysis was to evaluate the relationship between a treatment response biomarker and patient‐reported outcomes (PROs). Longitudinal data for PROs were obtained from adults with XLH from a phase III study.
Krina Mehta +6 more
wiley +1 more source
Orthodontics &Craniofacial Research, Volume 27, Issue 5, Page 697-703, October 2024.Abstract Objectives X‐linked hypophosphatemia (XLH) is a rare genetic disease that disturbs bone and teeth mineralization. It also affects craniofacial growth and patients with XLH often require orthodontic treatment. The aim of this study was to describe changes in the dental health of XLH children during orthodontic treatment compared with those in ...
Yann Janssens +5 more
wiley +1 more source
American Journal of Medical Genetics Part A, Volume 194, Issue 9, September 2024.Abstract Molecular genetics enables more precise diagnoses of skeletal dysplasia and other skeletal disorders (SDs). We investigated the clinical utility of multigene panel testing for 5011 unrelated individuals with SD in the United States (December 2019–April 2022).
Gretchen MacCarrick +13 more
wiley +1 more source
Safety and efficacy of burosumab in improving phosphate metabolism, bone health, and quality of life in adolescents with X-linked hypophosphatemic rickets [PDF]
Background and objective: X-linked hypophosphatemic rickets (XLH) is due to loss-of-function mutations in the phosphate-regulating endopeptidase homologue on the X chromosome (PHEX) that lead to increased fibroblast growth factor 23 (FGF23) production ...
Aversa Antonio +9 more
core +1 more source