Results 61 to 70 of about 1,121 (157)

Pharmacodynamic Exposure–Response Analysis of Fracture Count Data Following Treatment with Burosumab in Patients with XLH

The Journal of Clinical Pharmacology, Volume 65, Issue 2, Page 253-260, February 2025.
Abstract X‐linked hypophosphatemia (XLH) is a rare genetic disorder caused by excessive fibroblast growth factor 23 (FGF23), leading to low serum phosphate levels resulting in increased risk of fractures and pseudofractures. Burosumab is indicated for the treatment of XLH.
Krina Mehta, Jose Storopoli, Nikita Ramwani, Emilia Quattrocchi, Joga Gobburu, Thomas Weber, Matthew W. Hruska, Douglas Marsteller   +7 more
wiley   +1 more source

Navigating Recent Changes in Dosing Information: Dynamics of FDA‐Approved Monoclonal Antibodies in Post‐Marketing Realities

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, Su‐jin Rhee, Seong Min Koo, So Won Kim, Gyo Eun Lee, Yoon A Yie, Yun Kim   +6 more
wiley   +1 more source

Assessing Post‐Marketing Requirements for Orphan Drugs: A Cross‐Sectional Analysis of FDA and EMA Oversight

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, Sangwon Lee, Yoon Jung Kim, Won Young Kim, Min Jung Lee, Yun Kim   +5 more
wiley   +1 more source

Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients

Bone Reports
X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020.
Julia Day, Chandrin Jayatilleke, Matthew Roy   +2 more
doaj   +1 more source

Issue Information

Pediatric Discovery, Volume 4, Issue 2, June 2026.
No abstract is available for this article.
wiley   +1 more source

Real-world non-interventional post-authorization safety study of long-term use of burosumab in children and adolescents with X-linked hypophosphatemia: first interim analysis

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

Use of burosumab in McCune Albright syndrome: case report and review of literature in mosaic disorders with FGF23 overproduction

Frontiers in Endocrinology
Increased fibroblast growth factor 23 (FGF23) related mosaic syndromes include a spectrum of disorders sharing postzygotic mutations, skin involvement and dysplastic bone lesions.
Alessandro Barbato, Alessandro Barbato, Renato Vaiasuso, Renato Vaiasuso, Eugenio Trinati, Eugenio Trinati, Giulia Del Medico, Giulia Del Medico, Nicolò Chiti, Giampiero Igli Baroncelli, Stefano Stagi, Stefano Stagi   +11 more
doaj   +1 more source

Burosumab for the treatment of cutaneous-skeletal hypophosphatemia syndrome

Bone Reports
Cutaneous-skeletal hypophosphatemia syndrome (CSHS) is a rare bone disorder featuring fibroblast growth factor-23 (FGF23)-mediated hypophosphatemic rickets. We report a 2-year, 10-month-old girl with CSHS treated with burosumab, a novel human monoclonal antibody targeting FGF23.
Lillian Abebe, Kim Phung, Marie-Eve Robinson, Richelle Waldner, Sasha Carsen, Kevin Smit, Andrew Tice, Joanna Lazier, Christine Armour, Marika Page, Saunya Dover, Frank Rauch, Khaldoun Koujok, Leanne M. Ward   +13 more
openaire   +3 more sources

Effect of burosumab conversion on calciuria and nephrocalcinosis in children with XLH: A real-world cohort study

Bone Reports
Background: Burosumab, a monoclonal antibody to fibroblast growth factor 23 (FGF23), is effective for X-linked hypophosphatemic rickets (XLH). Renal effects, particularly calciuria and nephrocalcinosis, remain incompletely characterized. Methods: In this
Guido Filler, Harry Chandrakumaran, Funmbi Babalola, Dougenie Emile, Shih-Han Susan Huang, Robert Stein   +5 more
doaj   +1 more source

Nephrocalcinosis tendency does not worsen under burosumab treatment for X-linked hypophosphatemic rickets: a multicenter pediatric study

Frontiers in Pediatrics
BackgroundX-linked hypophosphatemic rickets (XLH) is associated with uninhibited FGF23 activity, which leads to phosphaturia, hypophosphatemia and depressed active vitamin D (1,25OH2D) levels. Conventional treatment with phosphate supplements and vitamin
Shelly Levi, Daniel Landau, Daniel Landau, Miriam Davidovits, Miriam Davidovits, Mika Shapira Rootman, Avivit Brener, Avivit Brener, Shoshana Gal, Shoshana Gal, Yael Borovitz, Yael Borovitz, Ori Goldberg, Ori Goldberg, Ori Goldberg, Rachel Bello, Roxana Cleper, Roxana Cleper, Yael Lebenthal, Yael Lebenthal, Yael Levy-Shraga, Yael Levy-Shraga, Dov Tiosano, Dov Tiosano, Adi Chezana, Ravit Regev, Ravit Regev, Leonid Zeitlin, Leonid Zeitlin   +28 more
doaj   +1 more source