Results 31 to 40 of about 937 (188)
Medical Management of Hypophosphatasia: Review of Data on Asfotase Alfa. [PDF]
Curr Osteoporos RepAbstract 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
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 +3 more
doaj +1 more source
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
JBMR Plus, 2021 Treatment with asfotase alfa has transformed the prognosis of hypophosphatasia in children and improves the bone and muscle signs in adults. The doses used in adults are the same as in children, whereas bone remodeling is different between them.
Michel Laroche +6 more
doaj +1 more source
There’s Something in the Water: An Unusual Case of Hypophosphatasia [PDF]
, 2023 Learning Objectives 1. Explain the mechanism of TNSALP in the pathogenesisof hypophosphatasia and the role of ALPL mutations inthe severity of disease presentation. 2.
Fan, Chris, Wills, Carson
core +2 more sources
ADULT-ONSET HYPOPHOSPHATASIA: BEFORE AND AFTER TREATMENT WITH ASFOTASE ALFA. [PDF]
AACE Clin Case Rep, 2019 To review the diagnosis and clinical course of a woman with hypophosphatasia who is being treated with newly approved enzyme replacement therapy, asfotase alfa.Clinical and laboratory data are presented.This is a unique report of a woman with debilitating adult-onset hypophosphatasia who was successfully diagnosed with low alkaline phosphatase (ALP ...
Magdaleno AL +4 more
europepmc +5 more sources
Dual X-ray absorptiometry has limited utility in detecting bone pathology in children with hypophosphatasia: A pooled post hoc analysis of asfotase alfa clinical trial data [PDF]
, 2020 Asfotase alfa is an enzyme replacement therapy approved for treatment of patients with pediatric-onset hypophosphatasia (HPP), a rare, inherited, systemic disease causing impaired skeletal mineralization, short stature, and reduced physical function in ...
Martos-Moreno, Gabriel +4 more
core +1 more source
Validation of a novel scoring system for changes in skeletal manifestations of hypophosphatasia in newborns, infants, and children: The Radiographic Global Impression of Change scale [PDF]
, 2018 Hypophosphatasia (HPP) is the heritable metabolic disease characterized by impaired skeletal mineralization due to low activity of the tissue-nonspecific isoenzyme of alkaline phosphatase.
Fujita, Kenji P +4 more
core +2 more sources
Clinical variability of hypophosphatasia in colombian patients : case reports [PDF]
, 2021 Q4Q4Pacientes con HipofosfatasiaHypophosphatasia (HPP) is a rare inherited disorder characterized by low serum alkaline phosphatase. It affects bone and tooth mineralization, although extra-skeletal manifestations are frequent.
Gonzáles López, Vladimir +5 more
core +1 more source