Effect of asfotase alfa in the treatment of hypophosphatasia- A systematic review
Journal of Pharmacy and Bioallied Sciences, 2023 Hypophosphatasia (HPP) is a life-threatening disease that occurs due to the mutation of the TNSALP (Tissue nonspecific isoenzyme of alkaline phosphatase) encoding gene. There is no approved treatment for Hypophosphatasia.
N Jaswanthi +7 more
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Radiological evaluation of pseudofracture after the administration of asfotase alfa in an adult with benign prenatal hypophosphatasia: A case report [PDF]
Bone Reports, 2022 Hypophosphatasia (HPP) is a congenital disorder with decreased activity of tissue-nonspecific alkaline phosphatase. Asfotase alfa is the only treatment approved for HPP and improves the impairment of bone mineralization. Although several previous studies
Hajime Kato, Naoko Hidaka, Minae Koga
exaly +4 more sources
Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia [PDF]
JIMD Reports, 2021 Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by deficient activity of alkaline phosphatase, causing defective mineralization of bones and teeth.
Olivia Sarah Strandbech +2 more
doaj +6 more sources
Case Report: Suboptimal response to standard-dose asfotase alfa in perinatal hypophosphatasia indicates a need for individualized dosing [PDF]
Frontiers in EndocrinologyHypophosphatasia (HPP) is a rare, inherited metabolic disorder due to a deficiency of tissue-nonspecific alkaline phosphatase, characterized by defective bone and teeth mineralization with consequent problems, including respiratory failure in severe ...
Danijela Petković Ramadža +7 more
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Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia [PDF]
Italian Journal of PediatricsBackground Hypophosphatasia (HPP) is a rare inherited disorder characterized by a deficiency of tissue-non-specific alkaline phosphatase (TNSALP) due to loss-of-function variants of the ALPL gene.
Debora Mariarita d’Angelo +10 more
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Fracture Healing in Two Adult Patients With Hypophosphatasia After Asfotase Alfa Therapy [PDF]
JBMR Plus, 2018 ABSTRACTInfants and children with hypophosphatasia (HPP) treated with asfotase alfa show improvement in bone mineralization and motor function, but it is unclear whether the medication can affect fracture healing in adult HPP patients. We present the course of fracture healing in two adults with HPP on enzyme replacement. Case 1 is a 41‐year‐old female
Steven W InG
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Altered Thyroid Function Tests Observed in Hypophosphatasia Patients Treated with Asfotase Alfa [PDF]
International Journal of Endocrinology, 2021 Background. Asfotase alfa is the only approved treatment that can normalize mineralization in patients with hypophosphatasia (HPP). Its interference in alkaline phosphatase (ALP) dependent immunoassays has been reported. Objective.
Hajime Kato +6 more
doaj +2 more sources
Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy [PDF]
Drug Design, Development and Therapy, 2018 Sasigarn A Bowden,1 Brian L Foster2 1Division of Endocrinology, Department of Pediatrics, Nationwide Children’s Hospital/The Ohio State University College of Medicine, Columbus, OH 43205, USA; 2Division of Biosciences, College of Dentistry, The ...
Bowden SA, Foster BL
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Experiences with the use of asfotase alfa in three patients with childhood-onset hypophosphatasia [PDF]
Endocrinology, Diabetes & Metabolism Case ReportsHypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by deficient activity of serum alkaline phosphatase due to loss-of-function mutations in the ALPL gene.
Francesca Pigliaru +2 more
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Hearing impairment improved after treatment with asfotase alfa in a case of perinatal hypophosphatasia [PDF]
Molecular Genetics and Metabolism Reports, 2020 Hearing impairment is a neurological symptom of hypophosphatasia (HPP), which leads to a reduced quality of life. However, the pathomechanism of hearing impairment and the effects of asfotase alfa enzyme replacement therapy on hearing function in HPP ...
Rie Chida-Naomiya +4 more
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