asfotase alfa

07/01/2017 - "Safety and efficacy of treatment with asfotase alfa in patients with hypophosphatasia: Results from a Japanese clinical trial."
10/01/2021 - "These data, together with previously published pharmacodynamic results in this study population, support the use of asfotase alfa at the recommended dose of 6 mg/kg/wk in adults with pediatric-onset hypophosphatasia."
01/01/2019 - "Letter to the Editor: "Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study"."
01/01/2019 - "Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study."
01/01/2019 - "The primary objectives of this extension study were to assess the long-term tolerability of asfotase alfa, defined as the number of patients with one or more treatment-emergent adverse events, and skeletal manifestations associated with hypophosphatasia, evaluated using the Radiographic Global Impression of Change (RGI-C) scale (-3 indicating severe worsening, and +3 complete or near-complete healing). "

01/01/2018 - "In conclusion, treatment with asfotase alfa significantly improved physical function, pain, overall quality of life, and skeletal radiographic findings in this patient. "
11/01/2019 - "Treatment with asfotase alfa resolved her chronic bony pain symptoms and quadrupled her daily pedometer step count. "
06/01/2022 - "Replacement therapy with asfotase alfa was started, resulting in pain relief in two months, and the disappearance of nuclide accumulation on bone scintigraphy and union of the pseudofracture on X-ray after two years. "
05/01/2016 - "In childhood HPP, asfotase alfa improved growth, gross motor function, strength and agility and decreased pain. "
11/01/2020 - "This study aims to assess patient-reported pain, disability and health-related quality of life (HRQoL) in a real-world cohort of adults with HPP who were not receiving asfotase alfa during the analysis. "

04/01/2020 - "Asfotase-alfa improves respiratory insufficiency, bone mineralization, and long-term survival, and has a very good safety profile."
12/01/2022 - "Enzyme replacement therapy (ERT) using asfotase alfa promptly improved her respiratory insufficiency, bone mineralization, and maintained her motor development during infancy. "
03/01/2023 - "Enzyme replacement therapy (asfotase alfa) has been shown to improve respiratory insufficiency and skeletal complications in HPP patients, while its effects on dental status have been understudied to date. "

03/08/2012 - "We enrolled infants and young children with life-threatening or debilitating perinatal or infantile hypophosphatasia in a multinational, open-label study of treatment with ENB-0040. "
01/01/2019 - "Patients with perinatal or infantile hypophosphatasia treated with asfotase alfa for up to 7 years showed early, sustained improvements in skeletal mineralisation. "
01/01/2019 - "Asfotase alfa treatment in perinatal and infantile hypophosphatasia: safe and sustained efficacy."
01/01/2016 - "Asfotase Alfa Treatment Improves Survival for Perinatal and Infantile Hypophosphatasia."
01/01/2019 - "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. "

01/01/2023 - "Overall, in this child with a compound pathogenic variant in PIGN, off-label use of asfotase alfa has been generally well tolerated with minimal side effects and resolution of rickets, but she continues to remain skeletally fragile."
02/01/2016 - "In noncomparative clinical trials in infants and children with paediatric-onset HPP, asfotase alfa rapidly improved radiographically-assessed rickets severity scores at 24 weeks (primary timepoint) as reflected in improvements in bone mineralization, with these benefits sustained after more than 3 years of treatment. "