A decade of FDA-approved drugs (2010–2019): trends and future directions

DG Brown, HJ Wobst - Journal of medicinal chemistry, 2021 - ACS Publications
A total of 378 novel drugs and 27 biosimilars approved by the US Food and Drug
Administration (FDA) between 2010 and 2019 were evaluated according to approval …

A clinician's guide to X‐linked hypophosphatemia

TO Carpenter, EA Imel, IA Holm… - Journal of Bone and …, 2011 - academic.oup.com
X‐linked hypophosphatemia (XLH) is the prototypic disorder of renal phosphate wasting,
and the most common form of heritable rickets. Physicians, patients, and support groups …

Effect of burosumab compared with conventional therapy on younger vs older children with X-linked hypophosphatemia

LM Ward, FH Glorieux, MP Whyte… - The Journal of …, 2022 - academic.oup.com
Context Younger age at treatment onset with conventional therapy (phosphate salts and
active vitamin D; Pi/D) is associated with improved growth and skeletal outcomes in children …

X-linked hypophosphatemia and FGF23-related hypophosphatemic diseases: prospect for new treatment

Y Kinoshita, S Fukumoto - Endocrine reviews, 2018 - academic.oup.com
Phosphate plays essential roles in many biological processes, and the serum phosphate
level is tightly controlled. Chronic hypophosphatemia causes impaired mineralization of the …

Nationwide survey of fibroblast growth factor 23 (FGF23)-related hypophosphatemic diseases in Japan: prevalence, biochemical data and treatment

I Endo, S Fukumoto, K Ozono, N Namba, D Inoue… - Endocrine …, 2015 - jstage.jst.go.jp
A nationwide epidemiologic survey of fibroblast growth factor 23 (FGF23)-related
hypophosphatemic diseases was conducted in 2010 to clarify the prevalence and the …

Transgenic mice overexpressing human fibroblast growth factor 23 (R176Q) delineate a putative role for parathyroid hormone in renal phosphate wasting disorders

X Bai, D Miao, J Li, D Goltzman, AC Karaplis - Endocrinology, 2004 - academic.oup.com
Abstract Fibroblast growth factor 23 (FGF23) is a recently characterized protein likely
involved in the regulation of serum phosphate homeostasis. Increased circulating levels of …

A translocation causing increased α-Klotho level results in hypophosphatemic rickets and hyperparathyroidism

CA Brownstein, F Adler… - Proceedings of the …, 2008 - National Acad Sciences
Phosphate homeostasis is central to diverse physiologic processes including energy
homeostasis, formation of lipid bilayers, and bone formation. Reduced phosphate levels due …

Conventional therapy in adults with X-linked hypophosphatemia: effects on enthesopathy and dental disease

J Connor, EA Olear, KL Insogna, L Katz… - The Journal of …, 2015 - academic.oup.com
Context: Treatment of X-linked hypophosphatemia (XLH) with active vitamin D metabolites
and phosphate can partially correct skeletal deformities. It is unclear whether therapy …

Burosumab: first global approval

YN Lamb - Drugs, 2018 - Springer
Abstract Burosumab (Crysvita®; Kyowa Hakko Kirin Co., Ltd. and Ultragenyx
Pharmaceutical Inc.) is a fully human monoclonal antibody directed at fibroblast growth …

Mutational analysis and genotype-phenotype correlation of the PHEX gene in X-linked hypophosphatemic rickets

IA Holm, AE Nelson, BG Robinson… - The Journal of …, 2001 - academic.oup.com
PHEX is the gene defective in X-linked hypophosphatemic rickets. In this study, analysis of
PHEX revealed mutations in 22 hypophosphatemic rickets patients, including 16 of 28 …