Results 31 to 40 of about 717 (150)

Bone turnover and mineral metabolism in adult patients with hypophosphatasia treated with asfotase alfa. [PDF]

Osteoporos Int, 2021
Summary There is limited understanding of how asfotase alfa affects mineral metabolism and bone turnover in adults with pediatric-onset hypophosphatasia.
Seefried L, Rak D, Petryk A, Genest F.
europepmc   +2 more sources

Asfotase-alfa [PDF]

Reactions Weekly, 2017
core   +5 more sources

HYPOPHOSPHATASIA IN ADULTS: CLINICAL CASES AND LITERATURE REVIEW [PDF]

Остеопороз и остеопатии, 2015
Hypophosphatasia is a rare inborn error of metabolism caused by mutations in the gene encoding tissue-nonspecific isoenzyme of alkaline phosphatase. Inexpressive clinical picture of the disease in adults is often difficult to diagnose.
S S Rodionova, E Yu Zakharova, Yu V Buklemishev, U R Khakimov, S V Lapkina   +4 more
doaj   +3 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

Asfotase alfa has a limited effect in improving the bowed limbs in perinatal benign hypophosphatasia: A case report [PDF]

Clinical Pediatric Endocrinology, 2021
Hiroshi Kitoh, Shiro Imagama, Kenichi Mishima   +2 more
exaly   +2 more sources

The Effect of Asfotase Alfa on Plasma and Urine Pyrophosphate Levels and Pseudofractures in a Patient With Adult-Onset Hypophosphatasia. [PDF]

JBMR Plus, 2023
Hypophosphatasia (HPP) is an inherited disease caused by variants of the ALPL gene encoding tissue‐nonspecific alkaline phosphatase. Adult‐onset HPP (adult HPP), known as a mild form of HPP, develops symptoms involving osteomalacia after the age of 18 ...
Hidaka N, Murata H, Tachikawa K, Osaki K, Sekiyama T, Kinoshita Y, Kato H, Hoshino Y, Kimura S, Sunouchi T, Watanabe S, Nangaku M, Makita N, Michigami T, Ito N.   +14 more
europepmc   +2 more sources

Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series

Bone Reports, 2022
Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61–68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15–48 months.
Cheryl Rockman-Greenberg, Robert Josse, Mira Francis, Aziz Mhanni   +3 more
doaj   +1 more source

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
Dahir KM, Dunbar NS.
europepmc   +3 more sources

Asfotase Alfa hypersensitivity: an outpatient 8-steps desensitization protocol [PDF]

Immunologic Research, 2021
Scudu S., Ghisu A., Costanzo G., Barca M. P., Firinu D., Del Giacco S.   +5 more
openaire   +4 more sources

Asfotase-alfa [PDF]

Reactions Weekly
core   +4 more sources