Results 51 to 60 of about 1,121 (157)
Hyperparathyroidism Secondary to Burosumab Treatment
CureusTumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome of abnormal phosphorus metabolism caused by increased secretion of fibroblast growth factor 23 (FGF23) by small mesenchymal tumors. In this article, we reported a patient with chronic refractory hypophosphatemia due to TIO who is treated with burosumab, a monoclonal antibody that ...
Nguyen, Minhtri K +2 more
openaire +2 more sources
Long-term use of burosumab for the treatment of tumor-induced osteomalacia
Osteoporosis International, 2022 Tumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome caused by tumoral overproduction of FGF-23. Due to local recurrence, we describe the long-term efficacy and safety profile of burosumab, an anti-FGF-23 monoclonal antibody, in a TIO patient after three unsuccessfully surgical attempts.TIO is a rare paraneoplastic syndrome caused by ...
Crotti, C +4 more
openaire +2 more sources
Informing Dose for Pediatric Rare Diseases—A Survey of Recent Orphan Drugs Approvals
Clinical and Translational Science, Volume 19, Issue 2, February 2026.ABSTRACT Collectively, pediatric rare diseases affect millions of children worldwide. Yet, treatment options are limited. Dose selection presents unique challenges in pediatric rare disease drug development. Traditional dose‐finding approaches are impractical for these populations, and conventional pediatric dosing methods like exposure matching face ...
Elimika Pfuma Fletcher +6 more
wiley +1 more source
Advanced Science, Volume 13, Issue 4, 19 January 2026.The pathogenic role of PHEX isn't fully determined, and there is no radical cure for X‐linked hypophosphatemic rickets (XLHR). This study makes the first attempt to perform gene therapy using a minicircle DNA (MC‐DNA) vector expressing a fragment of FGF23 (amino acids 180‐251) in Phex‐T1349C mice and suggests MC‐DNA as a promisingly safe and effective ...
Huixiao Wu +20 more
wiley +1 more source
X‐Linked Hypophosphatemia Caused by a New Partial Insertion of LINE‐1 in the PHEX Gene
Human Mutation, Volume 2026, Issue 1, 2026.X‐linked hypophosphatemia (XLH), primarily caused by mutations of the PHEX gene, is the most common cause of genetic rickets. Pediatric cases of XLH typically present with elevated levels of serum fibroblast growth factor 23 (FGF23), hypophosphatemia, rickets, and impaired growth.
Dongmei Li +6 more
wiley +1 more source
Burosumab Therapy in Children with X-Linked Hypophosphatemia
Yearbook of Paediatric Endocrinology, 2018 X-linked hypophosphatemia is characterized by increased secretion of fibroblast growth factor 23 (FGF-23), which leads to hypophosphatemia and consequently rickets, osteomalacia, and skeletal deformities. We investigated burosumab, a monoclonal antibody that targets FGF-23, in patients with X-linked hypophosphatemia.In an open-label, phase 2 trial, we ...
Carpenter, Thomas O. +13 more
openaire +4 more sources
The Journal of Clinical Pharmacology, Volume 65, Issue 12, Page 1889-1899, December 2025.Abstract In Japan, the percentage of approved drugs with pediatric indications increased to 30% in 2010‐2015, but no further increase was observed through 2020. The Ministry of Health, Labor, and Welfare in Japan presented draft future directions to promote pediatric drug development, where the modeling and simulation (M&S) approach was introduced as a
Akinori Nakashima +5 more
wiley +1 more source
Health Science Reports, Volume 8, Issue 9, September 2025.ABSTRACT Background and Aims Polycystic ovary syndrome (PCOS) is a prevalent endocrine and metabolic disorder with complex pathogenesis and limited targeted therapeutic options. This study presents a proteomics‐informed approach to identify potential plasma protein targets for PCOS intervention using causal inference methods.
Tong Yu, Pengfei Zeng, Hang Zhou
wiley +1 more source
Phosphate in Physiological and Pathological Mineralization: Important yet Often Unheeded
MedComm, Volume 6, Issue 7, July 2025.Phosphate serves as a building block for physiological mineralization, and as a signaling molecule that regulates the activity of mineralizing cells. The disturbance in these processes could induce a series of pathological mineralization, with abnormal mineralization of hard tissues and ectopic mineralization of soft tissues being the most ...
Wen Qin +8 more
wiley +1 more source
X-linked hypophosphatemic rickets: case report with perspectives and challenges [PDF]
Einstein (São Paulo)X-linked dominant hypophosphatemic rickets (XLH) is a rare genetic disorder caused by mutations in the PHEX gene and characterized by chronic hypophosphatemia due to dysregulation of fibroblast growth factor 23 (FGF23).
Beatriz Almeida Ribeiro +6 more
doaj +2 more sources