Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23

Imel, Erik A.; Zhang, Xiaoping; Ruppe, Mary D.; Weber, Thomas J.; Klausner, Mark A.; Itô, Takahiro; Vergeire, Maria; Humphrey, Jeffrey S.; Glorieux, Francis H.; Portale, Anthony A.; Insogna, Karl L.; Peacock, Munro; Carpenter, Thomas O.

doi:10.1210/jc.2015-1551

Cited by 146 publications

(143 citation statements)

References 25 publications

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“…However, insufficient treatment can in turn lead to complications such as bone fractures, which impact quality of life [2]. Recently, however, a new therapy using anti-FGF23 antibody has been tested [13,14], and hexa-Darginine was also reported as a new therapeutic agent [15], holding out the hope of the development of new methods of treatment with fewer complications.…”

Section: Discussionmentioning

confidence: 99%

Hypertension is a characteristic complication of X-linked hypophosphatemia

et al. 2017

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Section: Discussionmentioning

confidence: 99%

Hypertension is a characteristic complication of X-linked hypophosphatemia

et al. 2017

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“…Actually, several preclinical studies indicated that the inhibition of FGF23 activities may be useful for hypophosphatemic diseases caused by excessive actions of FG23 [3][4][5][6][7][8][9]. In addition, the results of phase I-II clinical trials of neutralizing anti-FGF23 antibody were published [10,11]. In this review, physiological actions of FGF23-FGF receptor/Klotho pathway and the possible ways to modulate this pathway are summarized.…”

Section: Introductionmentioning

confidence: 99%

FGF23-FGF Receptor/Klotho Pathway as a New Drug Target for Disorders of Bone and Mineral Metabolism

Fukumoto

2015

Calcif Tissue Int

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Fibroblast growth factor 23 (FGF23) is a phosphaturic hormone produced by bone and works by binding to Klotho-FGF receptor complex. Excessive and deficient actions of FGF23 result in hypophosphatemic and hyperphosphatemic diseases, respectively. Therefore, it is reasonable to think that modulating FGF23 activities may be a novel therapeutic measure for these diseases. Several preclinical reports indicate that the inhibition of FGF23 activities ameliorates hypophosphatemic rickets/osteomalacia caused by excessive actions of FGF23. In addition, phase I-II clinical trials of anti-FGF23 antibody in adult patients with X-linked hypophosphatemia rickets, the most prevalent cause of genetic FGF23-related hypophosphatemic rickets, indicated that the antibody enhances renal tubular phosphate reabsorption and increases serum phosphate. However, it is not known whether the inhibition of FGF23 activities actually brings clinical improvement of rickets and osteomalacia. Available data indicate that FGF23-FGF receptor/Klotho pathway can be a new drug target for disorders of phosphate and bone metabolism.

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“…У пациентов с Х-сцепленной фосфопенической остеомаляцией буросумаб повышает реабсорбцию фосфо-ра в почках и экспрессию фермента 1α-гидроксилазы, от-ветственного за метаболизм витамина D. Следует отметить, что при применении буросумаба не отмечалось развития вторичного гиперпаратиреоза и нефрокальциноза [18], в отличие от традиционной схемы консервативной терапии. Применение антитела к ФРФ23 на мышиной модели способ-ствовало обратному развитию деформаций скелета [29].…”

Section: Discussionunclassified

Rare genetic diseases of the bone tissue: the case of a family with osteogenesis imperfecta and X-linked hypophosphataemia

Popova

Юрьевна²,

Grebennikova

et al. 2018

Osteopor Bone Dis

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Osteogenesis imperfecta (OI) and X-linked hypophosphataemia (XLH) are rare genetic diseases, which lead to childhood-onset bone fragility, low-trauma fractures and limb deformities. OI occurs as a result of impaired type 1 collagen synthesis at different stages, depending on the type of a genetic mutation, which leads to bone strength impairment. In most cases OI is a disorder with an autosomal dominant inheritance. However, there are also cases of autosomal recessive inheritance. To date, 16 types of OI are distinguished, with type 2 being the most severe due to 100% mortality rate in neonatal and perinatal periods. XLH is characterized by altered bone mineralization due to impaired phosphorus absorption and reabsorption, as a result of mutations in the PHEX gene. The bone tissue «softens», and this process is accompanied by deformities in long tubular bones. In this article we describe the family, in which both diseases are presented, despite their rarity. The case is investigated from points of view: the clinician’s and the patient’s perspective.

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Prolonged Correction of Serum Phosphorus in Adults With X-Linked Hypophosphatemia Using Monthly Doses of KRN23

Abstract: Monthly KRN23 significantly increased serum Pi, TmP/GFR, and 1,25(OH)2D in all subjects. KRN23 has potential for effectively treating XLH.

Cited by 146 publications

References 25 publications

Hypertension is a characteristic complication of X-linked hypophosphatemia

Hypertension is a characteristic complication of X-linked hypophosphatemia

FGF23-FGF Receptor/Klotho Pathway as a New Drug Target for Disorders of Bone and Mineral Metabolism

Rare genetic diseases of the bone tissue: the case of a family with osteogenesis imperfecta and X-linked hypophosphataemia

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