Effect of asfotase alfa in the treatment of hypophosphatasia- A systematic review [PDF]
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
doaj +5 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 +8 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
exaly +7 more sources
Improvements in Bone Disorganization and Pseudo-Fracture Healing in Hypophosphatasia Following Asfotase Alfa Therapy May Be Detectable by the ALIGNOGRAM Before Changes in Bone Radiography or Scintigraphy [PDF]
Case Reports in EndocrinologyHypophosphatasia (HPP) is a rare genetic disorder characterized by bone fragility due to defective bone mineralization resulting in impaired alignment of bone components (bone disorganization).
Roger Zebaze +5 more
doaj +5 more sources
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 +3 more sources
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
Masaomi Nangaku +2 more
exaly +4 more sources
Efficacy of asfotase alfa in a patient with adult-onset hypophosphatasia without obvious bone lesions: a case report with review of literature [PDF]
Endocrine JournalThe use of asfotase alfa, a bone-targeted recombinant alkaline phosphatase (ALP) enzyme, for the treatment of adult-onset hypophosphatasia (HPP) remains controversial, particularly in patients without evident bone abnormalities.
Seiji Nishikage +11 more
doaj +4 more sources
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
doaj +2 more sources
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
doaj +2 more sources
A two-year follow-up of asfotase alfa replacement in a patient with hypophosphatasia: clinical, biochemical, and radiological evaluation [PDF]
Archives of Endocrinology and Metabolism, 2020 SUMMARY Hypophosphatasia (HPP) is a rare disease with a high mortality rate in its severe forms. It is caused by mutations within the gene encoding the tissue-nonspecific alkaline phosphatase (TNSALP), an enzyme responsible for bone mineralization.
Fernanda Salles Reis +3 more
doaj +2 more sources