Visualization of asfotase alfa-binding to sites of calcification in vivo
Bone Reports, 2020 Flavia Amadeu de Oliveira +3 more
doaj +2 more sources
Effect of Asfotase Alfa on Muscle Weakness in a Japanese Adult Patient of Hypophosphatasia with Low ALP Levels [PDF]
Internal Medicine, 2020 Yasuhisa Ohata, Kenro Imaeda
exaly +2 more sources
Asfotase alfa improved skeletal mineralization and fracture healing in a child with MCAHS. [PDF]
Bone, 2023 Tissue non-specific alkaline phosphatase (TNSALP) is an enzyme that is tethered to the cell membrane by glycosylphosphatidylinositol (GPI) and converts inorganic pyrophosphate to inorganic phosphate. Inorganic phosphate combines with calcium to form hydroxyapatite, the main mineral in the skeleton.
Kang M, Wu M, Crane JL.
europepmc +3 more sources
Hypophosphatasia: from birth to adulthood [PDF]
Archives of Endocrinology and Metabolism, 2023 Hypophosphatasia (HPP) is an inherited disease caused by a low activity of tissue-nonspecific alkaline phosphatase, a hydrolase that removes phosphate groups from many molecules.
Fernanda Salles Reis +1 more
doaj +1 more source
Improvement in quality of life after asfotase alfa treatment in adults with pediatric-onset hypophosphatasia: data from 5 patient-reported outcome measures [PDF]
JBMR PlusSteven W InG +2 more
exaly +2 more sources
Characterization of tracheobronchomalacia in infants with hypophosphatasia
Orphanet Journal of Rare Diseases, 2020 Background Perinatal and infantile hypophosphatasia (HPP) are associated with respiratory failure and respiratory complications. Effective management of such complications is of key clinical importance.
Raja Padidela +12 more
doaj +1 more source
Pharmacodynamics of asfotase alfa in adults with pediatric-onset hypophosphatasia
Bone, 2021 Hypophosphatasia (HPP) is the rare, inherited, metabolic bone disease characterized by low activity of the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP) leading to excess extracellular inorganic pyrophosphate (PPi) and pyridoxal 5'-phosphate (PLP). Asfotase alfa is the human recombinant enzyme-replacement therapy that replaces deficient
Lothar, Seefried +6 more
openaire +2 more sources
Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia [PDF]
, 2019 Hypophosphatasia (HPP) features low tissue-nonspecific alkaline phosphatase (TNSALP) isoenzyme activity resulting in extracellular accumulation of its substrates including pyridoxal 5\u27-phosphate (PLP), the principal circulating form of vitamin B6, and
Watsky, Eric +7 more
core +2 more sources
Additional file 1 of A Japanese single-center experience of the efficacy and safety of asfotase alfa in pediatric-onset hypophosphatasia [PDF]
, 2022 Additional file 1. Video record of patient 2 demonstrating the patient’s inability to walk and the subsequent improvement in pain and walking ability after asfotase alfa (AA) initiation.
Taijiro Watanabe (12129659) +11 more
core +1 more source
A Rare Case of Perinatal Hypophosphatasia Treated With Asfotase Alfa [PDF]
J Endocr Soc, 2021 Abstract Background: Perinatal Hypophosphatasia (HPP) is a rare and lethal disorder associated with a 50–100% mortality rate, usually due to respiratory complications. HPP occurs due to a loss-of-function mutation in the ALPL gene, responsible for the function of tissue-nonspecific alkaline phosphatase (TNSALP).
Srivastava P +4 more
europepmc +3 more sources