Results 31 to 40 of about 1,121 (157)

Growth pattern in children with X-linked hypophosphatemia treated with burosumab and growth hormone

Orphanet Journal of Rare Diseases, 2022
Background X-linked hypophosphatemia (XLH) is characterized by increased serum concentrations of fibroblast growth factor 23 (FGF23), hypophosphatemia and insufficient endogenous synthesis of calcitriol.
Diana-Alexandra Ertl, Justin Le Lorier, Andreas Gleiss, Séverine Trabado, Candace Bensignor, Christelle Audrain, Volha Zhukouskaya, Régis Coutant, Jugurtha Berkenou, Anya Rothenbuhler, Gabriele Haeusler, Agnès Linglart   +11 more
doaj   +1 more source

Prediction of the structural interface between fibroblast growth factor23 and Burosumab using alanine scanning and molecular docking

Scientific Reports, 2022
Burosumab, an FGF23 targeting monoclonal antibody, was approved by the FDA in 2018 for use in children and adults with X-linked hypophosphatemia (or XLH).
Karnpob Kanhasut, Kannan Tharakaraman, Mathuros Ruchirawat, Jutamaad Satayavivad, Mayuree Fuangthong, Ram Sasisekharan   +5 more
doaj   +1 more source

Anticipated effects of burosumab treatment on long-term clinical sequelae in XLH: expert perspectives

Frontiers in Endocrinology, 2023
X-linked hypophosphatemia (XLH) is a rare, progressive, genetic disease with multisystem impact that typically begins to manifest in early childhood. Two treatment options exist: oral phosphate in combination with active vitamin D (“conventional therapy”)
Lothar Seefried, Martin Biosse Duplan, Martin Biosse Duplan, Martin Biosse Duplan, Karine Briot, Michael T. Collins, Rachel Evans, Pablo Florenzano, Pablo Florenzano, Neil Hawkins, Muhammad Kassim Javaid, Robin Lachmann, Leanne M. Ward   +12 more
doaj   +1 more source

What are the benefits of the anti-FGF23 antibody burosumab on the manifestations of X-linked hypophosphatemia in adults in comparison with conventional therapy? A review

Therapeutic Advances in Rare Disease, 2022
X-linked hypophosphatemia (XLH) is a genetic disease mostly related to PHEX gene mutations which increases FGF23 serum levels, leading to hypophosphatemia and osteomalacia in adults, while affected children, in addition, develop rickets.
Marie-Hélène Lafage-Proust
doaj   +1 more source

Treatment of X-Linked Hypophosphatemia in Children

Endocrines, 2022
The conventional treatment for X-linked hypophosphatemia (XLH), consisting of phosphorus supplementation and a biologically active form of vitamin D (alfacalcidol or calcitriol), is used to treat rickets and leg deformities and promote growth.
Toshihiro Tajima, Yukihiro Hasegawa
doaj   +1 more source

Burosumab for the Treatment of Tumor-Induced Osteomalacia [PDF]

Journal of Bone and Mineral Research, 2020
ABSTRACT Tumor-induced osteomalacia (TIO) is caused by phosphaturic mesenchymal tumors producing fibroblast growth factor 23 (FGF23) and is characterized by impaired phosphate metabolism, skeletal health, and quality of life. UX023T-CL201 is an ongoing, open-label, phase 2 study investigating the safety and efficacy of burosumab, a ...
Suzanne M Jan de Beur, Paul D Miller, Thomas J Weber, Munro Peacock, Karl Insogna, Rajiv Kumar, Frank Rauch, Diana Luca, Tricia Cimms, Mary Scott Roberts, Javier San Martin, Thomas O Carpenter   +11 more
openaire   +2 more sources

Positive Response to One-Year Treatment With Burosumab in Pediatric Patients With X-Linked Hypophosphatemia

Frontiers in Pediatrics, 2020
X-linked hypophosphatemia (XLH) causes significant burden in pediatric patients in spite of maintained treatment with phosphate supplements and vitamin D derivatives.
Silvia Martín Ramos, Marta Gil-Calvo, Virginia Roldán, Ana Castellano Martínez, Fernando Santos, Fernando Santos, Fernando Santos   +6 more
doaj   +1 more source

Combined treatment by burosumab and a calcimimetic can ameliorate hypophosphatemia due to excessive actions of FGF23 and PTH in adult XLH with tertiary hyperparathyroidism: A case report

Frontiers in Endocrinology, 2022
IntroductionX-linked hypophosphatemia (XLH) is the most prevalent type of heritable fibroblast growth factor 23 (FGF23)-related hypophosphatemic rickets. Recently, anti-FGF23 antibody, burosumab, has become clinically available.
Yuichi Takashi, Kyoko Toyokawa, Naoki Oda, Yoshimi Muta, Hisashi Yokomizo, Seiji Fukumoto, Daiji Kawanami   +6 more
doaj   +1 more source

Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment

RMD Open, 2023
Objectives To report the impact of continued burosumab treatment on clinical laboratory tests of efficacy, patient-reported outcomes (PROs) and ambulatory function in adults with X-linked hypophosphataemia who continued from a 96-week phase 3 study into ...
Karine Briot, Wei Sun, Rachel K Crowley, Maria Luisa Brandi, Stuart H Ralston, Peter Kamenický, Martine Cohen-Solal, Richard Keen, Angela Williams, Muhammad K Javaid, Robin H Lachmann, Annabel Nixon, Mark Nixon, Anne-Lise Lecoq, Sami Kolta, Jennifer S Walsh, Angela J Rylands   +16 more
doaj   +1 more source

Post-authorisation safety study of burosumab use in paediatric, adolescent and adult patients with X-linked hypophosphataemia: rationale and description

Therapeutic Advances in Chronic Disease, 2022
Background: X-linked hypophosphataemia (XLH) is a rare, inherited, phosphate-wasting disorder that elevates fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D (1,25(OH) 2 D) synthesis.
Maria Luisa Brandi, Gema Ariceta, Signe Sparre Beck-Nielsen, Annemieke M. Boot, Karine Briot, Carmen de Lucas Collantes, Francesco Emma, Sandro Giannini, Dieter Haffner, Richard Keen, Elena Levtchenko, Outi Mӓkitie, Ola Nilsson, Dirk Schnabel, Liana Tripto-Shkolnik, M. Carola Zillikens, Jonathan Liu, Alina Tudor, M. Zulf Mughal   +18 more
doaj   +1 more source