Results 31 to 40 of about 1,955 (223)

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

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 in Unresectable/Unidentifiable Tumour-Induced Osteomalacia [PDF]

Dr. Yi Shan Der, Dr. Santosh Chaubey, Dr. Anna McLean, Dr. Yasmin Trinh, Ms. Shelley Pember, Dr. Ashim Sinha   +5 more
openaire   +2 more sources

Sustained Efficacy and Safety of Burosumab, a Monoclonal Antibody to FGF23, in Children With X-Linked Hypophosphatemia [PDF]

, 2021
PURPOSE: In X-linked hypophosphatemia (XLH), excess fibroblast growth factor-23 causes hypophosphatemia and low calcitriol, leading to musculoskeletal disease with clinical consequences.
Boot, Annemieke M., Carpenter, Thomas O., Chen, Angel, Gottesman, Gary S., Hogler, Wolfgang, Imel, Erik A., Linglart, Agnes, Padidela, Raja, Portale, Anthony A., Roberts, Mary Scott, Skrinar, Alison, Van't Hoff, William, Whyte, Michael P.   +12 more
core   +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

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

A case report to assess the safety and efficacy of Burosumab, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome and associated hypophosphatemic rickets

Bone Reports, 2022
Epidermal Nevus Syndrome (ENS), also known as Cutaneous Skeletal Hypophosphatemia Syndrome or Linear Sebaceous Nevus Syndrome, is caused by a mosaic somatic mutation of RAS (Rat Sarcoma genes) which leads to abnormally elevated levels of fibroblast ...
Carson Huynh, Andrea Gillis, Jessica Fazendin, Hussein Abdullatif   +3 more
doaj   +1 more source

Patient-reported outcomes from a randomized, active-controlled, open-label, phase 3 trial of burosumab versus conventional therapy in children with X-linked hypophosphatemia [PDF]

, 2021
Changing to burosumab, a monoclonal antibody targeting fibroblast growth factor 23, significantly improved phosphorus homeostasis, rickets, lower-extremity deformities, mobility, and growth versus continuing oral phosphate and active vitamin D ...
Biggin, Andrew, Chen, Angel, Cheong, Hae Il, Glorieux, Francis H, Gottesman, Gary, Högler, Wolfgang, Imel, Erik, Munns, Craig F, Muroya, Koji, Namba, Noriyuki, Nilsson, Ola, Nixon, Annabel, Padidela, Raja, Perwad, Farzana, Pitukcheewanont, Pisit, Portale, Anthony, Simmons, Jill, Skrinar, Alison, Sochett, Etienne, Sun, Wei, Tanaka, Hiroyuki, Ward, Leanne M, Whyte, Michael P, Williams, Angela   +23 more
core   +5 more sources

A Case Report: First Long-Term Treatment With Burosumab in a Patient With Cutaneous-Skeletal Hypophosphatemia Syndrome

Frontiers in Endocrinology, 2022
Epidermal nevus syndromes encompass a highly heterogeneous group of systemic disorders, characterized by epidermal nevi, and a spectrum of neuromuscular, ocular, and bone abnormalities.
Lea Maria Merz, Florian Buerger, Niels Ziegelasch, Martin Zenker, Ilse Wieland, Tobias Lipek, Tillmann Wallborn, Nicolas Terliesner, Freerk Prenzel, Manuela Siekmeyer, Katalin Dittrich   +10 more
doaj   +1 more source

Phenotypic characterization of X-linked hypophosphatemia in pediatric Spanish population [PDF]

, 2021
Deformitats òssies; Retard en el creixement; Hipofosfatèmia hereditàriaDeformidades óseas; Retraso en el crecimiento; Hipofosfatemia hereditariaBone deformities; Growth retardation; Inherited hypophosphatemiaBackground X-linked hypophosphatemia (XLH) is
Ariceta Iraola, Gema, Chocron de Benzaquen, Sara, de la Cerda‑Ojeda, Francisco, Fernández‑Fernández, Marta, Gil‑Peña, Helena, Madariaga, Leire, Rodríguez‑Rubio, Enrique   +6 more
core   +4 more sources