Results 11 to 20 of about 948 (174)

Interference of Asfotase alfa in immunoassays employing alkaline phosphatase technology [PDF]

The Journal of Applied Laboratory Medicine, 2020
BACKGROUND: Asfotase alfa (STRENSIQ®, Alexion Pharmaceuticals, Inc.) is the only approved treatment for patients with pediatric-onset hypophosphatasia, a disease caused by a mutation in the tissue-nonspecific alkaline phosphatase (TNSALP) gene.
Fraser, William Duncan, Piec, Isabelle Danielle, Tompkins, Beatrice   +2 more
core   +8 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 Endocrinology
Hypophosphatasia (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, Simon Zhang, Cat Shore-Lorenti, Cherie Chiang, Frances Milat, Peter Ebeling   +5 more
doaj   +3 more sources

Mobility and Quality of Life in Adults with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement. [PDF]

Adv Ther
INTRODUCTION: Hypophosphatasia (HPP) is a rare disease caused by deficient tissue-non-specific alkaline phosphatase (ALP) activity. Asfotase alfa is a tissue-non-specific ALP enzyme-replacement therapy which was reimbursed in the UK under a Managed ...
Moss KE, Keen R, Fang S, Zygouras A, Javaid MK, Geberhiwot T, Poole KES, Poole KES, Selby P, Walsh JS, Bubbear JS.   +10 more
europepmc   +9 more sources

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
Klidaras P, Severt J, Aggers D, Payne J, Miller PD, Ing SW.   +5 more
europepmc   +6 more sources

Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study. [PDF]

J Clin Endocrinol Metab, 2019
Context Long-term data on enzyme replacement treatment of hypophosphatasia (HPP) are limited. Objective To evaluate efficacy and safety of asfotase alfa in patients aged ≤5 years with HPP followed for up to 6 years.
Hofmann CE, Harmatz P, Vockley J, Högler W, Nakayama H, Bishop N, Martos-Moreno GÁ, Moseley S, Fujita KP, Liese J, Rockman-Greenberg C, ENB-010-10 Study Group.   +11 more
europepmc   +7 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, Masaru Shimura, Ryuhei Nagao, Atsushi Kumada, Hisashi Kawashima   +4 more
doaj   +2 more sources

Pharmacokinetics of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia. [PDF]

J Clin Pharmacol, 2021
AbstractHypophosphatasia is a rare metabolic disease resulting from variant(s) in the gene‐encoding tissue‐nonspecific isozyme of alkaline phosphatase. In this 13‐week, phase 2a, multicenter, randomized, open‐label, dose‐response study (ClinicalTrials.gov: NCT02797821), the pharmacokinetics of asfotase alfa, an enzyme replacement therapy approved for ...
Pan WJ, Pradhan R, Pelto R, Seefried L.
europepmc   +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 Journal
The 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, Masaaki Yamamoto, Takahiro Niikura, Yuiko Inaba, Tomoyuki Akiyama, Risa Harada, Yoshitada Sakai, Kenji Sugawara, Kanako Tachikawa, Toshimi Michigami, Wataru Ogawa, Hidenori Fukuoka   +11 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, Débora Cristiane Gomes, Henrique Pierotti Arantes, Marise Lazaretti-Castro   +3 more
doaj   +2 more sources

Medical Management of Hypophosphatasia: Review of Data on Asfotase Alfa. [PDF]

Curr Osteoporos Rep
Abstract    Purpose Hypophosphatasia (HPP) is a rare, dento-osseous disorder caused by impaired activity of tissue non-specific alkaline phosphatase (TNSALP), a key enzyme in tissue mineralization. This review provides a clinical perspective on the current medical treatment of both children and adults with HPP.
Dahir KM, Dunbar NS.
europepmc   +3 more sources