Results 61 to 70 of about 1,955 (223)
Bone ReportsMany adolescents with X-linked hypophosphatemia (XLH) currently have to stop treatment with burosumab at the end of skeletal growth. We describe the experience of a cohort of adolescents with XLH before, during, and after stopping burosumab (median ...
Charlotte Jarvis +9 more
doaj +1 more source
Management of X-linked hypophosphatemic rickets: a review [PDF]
, 2020 There are two types of management in X-linked hypophosphatemic rickets (XLH), out of which the main stay of treatment is conventional treatment which includes combination of oral phosphate supplements and active vitamin D (calcitriol or alfacalcidol ...
Azar, Mohammed
core +2 more sources
The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data [PDF]
, 2023 Fibroblast growth factor 23; Patient registry; X-linked hypophosphatemiaFactor de creixement de fibroblasts 23; Registre de pacients; Hipofosfatèmia lligada a XFactor de crecimiento de fibroblastos 23; Registro de pacientes; Hipofosfatemia ligada al ...
Ariceta Iraola, Gema +5 more
core +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
An Expert Perspective on Phosphate Dysregulation With a Focus on Chronic Hypophosphatemia [PDF]
, 2020 Because of their rarity, diseases characterized by chronic hypophosphatemia can be underrecognized and suboptimally managed, resulting in poor clinical outcomes. Moreover, serum phosphate may not be measured routinely in primary care practice.
Aljuraibah, Fahad +11 more
core +1 more source
New treatments for rare bone diseases: hypophosphatemic rickets/osteomalacia
Archives of Endocrinology and Metabolism, 2022 Phosphorus is one of the most abundant minerals in the human body; it is required to maintain bone integrity and mineralization, in addition to other biological processes.
Julia Vieira Oberger Marques +2 more
doaj +1 more source
Epidemiology of Tumor-Induced Osteomalacia in Germany Based on Real World Data [PDF]
, 2023 Tumor-induced osteomalacia (TIO) is an ultra-rare disease caused mostly by benign tumors that secrete fibroblast growth factor-23. Because of nonspecific symptoms, the diagnostic delay is long, and therapy can be challenging.
Abrahamsen, Bo +8 more
core +1 more source
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
Impact of burosumab on lower limb alignment in children with X-linked hypophosphatemia
Journal of the Pediatric Orthopaedic Society of North America: Background: Osteotomy and hemiepiphysiodesis are used to treat lower limb deformities in the rare musculoskeletal disease X-linked hypophosphatemia (XLH), but postsurgical complications and malalignment recurrence are possible.
David B. Frumberg, MD, FAAOS +3 more
doaj +1 more source
Life, 2021 The authors present a stereotypical case presentation of X-linked hypophosphatemia (XLH) and provide a review of the pathophysiology and related pharmacology of this condition, primarily focusing on the FDA-approved medication burosumab.
Jon Vincze +5 more
doaj +1 more source