Asfotase Alfa Treatment in a 2-year-old Girl with Childhood Hypophosphatasia
Journal of Pediatric Research, 2022 Childhood hypophosphatasia (HPP) presents with bowing of the limbs, poor mobility, chronic pain, short stature, fractures, and motor impairment. Enzyme replacement therapy (ERT) provides improved pulmonary and physical function in life-threatening ...
Gönül Çatlı +4 more
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Mobility and Quality of Life in Children with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement. [PDF]
Adv TherIntroduction Hypophosphatasia (HPP) is a rare, inherited metabolic bone disease with a high degree of morbidity and mortality in children. Asfotase alfa is an enzyme replacement therapy for HPP reimbursed in the UK since 2017 under a Managed Access ...
Padidela R +7 more
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Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia [PDF]
Bone, 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
Bhatti, M Tariq +7 more
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Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. [PDF]
Lancet Diabetes and Endocrinology,the, 2019 BACKGROUND: Our previous phase 2, open-label study of 11 infants and young children with life-threatening perinatal or infantile hypophosphatasia showed 1 year safety and efficacy of asfotase alfa, an enzyme replacement therapy.
Balasubramaniam +29 more
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Phenotype and genotype of hypophosphatasia cases in Saudi Arabia: multi-center case cohort [PDF]
Frontiers in GeneticsIntroductionHypophosphatasia (HPP) is a rare inherited metabolic disease caused by mutations in the ALPL gene. The disease is heterogeneous, complicating its diagnosis and delaying optimal management, leading to severe or lethal outcomes such as failure ...
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Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy [PDF]
Drug Design, Development and Therapy, 2018 Sasigarn A Bowden,1 Brian L Foster2 1Division of Endocrinology, Department of Pediatrics, Nationwide Children’s Hospital/The Ohio State University College of Medicine, Columbus, OH 43205, USA; 2Division of Biosciences, College of Dentistry, The ...
Bowden SA, Foster BL
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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
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Bone turnover and mineral metabolism in adult patients with hypophosphatasia treated with asfotase alfa. [PDF]
Osteoporos Int, 2021 Seefried L, Rak D, Petryk A, Genest F.
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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
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