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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Effectiveness and safety of asfotase alfa for people with hypophosphatasia: a plain language summary of three studies [PDF]
Journal of Comparative Effectiveness ResearchWhat is this summary about? Hypophosphatasia (HPP for short) is a rare inherited condition that can present at any stage of life, with symptoms typically being more severe in cases that manifest earlier, such as during infancy or childhood.
Fatma Al Jasmi, Zhanna Belay
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Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry [PDF]
Orphanet Journal of Rare DiseasesBackground Hypophosphatasia (HPP) is a rare inherited disease caused by deficient activity of tissue-nonspecific alkaline phosphatase. Many adults with HPP have a high burden of disease, experiencing chronic pain, fatigue, limited mobility, and dental ...
Priya S. Kishnani +10 more
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Pathophysiology of hypophosphatasia and the potential role of asfotase alfa [PDF]
Therapeutics and Clinical Risk Management, 2016 Hideo Orimo Division of Metabolism and Nutrition, Department of Biochemistry and Molecular Biology, Nippon Medical School, Tokyo, Japan Abstract: Hypophosphatasia (HPP) is an inherited systemic bone disease that is characterized by bone ...
Orimo H
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Physical Function and Health‐Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric‐Onset Hypophosphatasia [PDF]
JBMR Plus, 2020 Hypophosphatasia (HPP) is a rare, inherited, metabolic disease characterized by tissue‐nonspecific alkaline phosphatase deficiency resulting in musculoskeletal and systemic clinical manifestations.
Franca Genest +3 more
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Asfotase Alfa Treatment in a 2-year-old Girl with Childhood Hypophosphatasia [PDF]
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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Effects of asfotase alfa on fracture healing of adult patient with hypophosphatasia and literature review [PDF]
Orphanet Journal of Rare DiseasesObjective Hypophosphatasia (HPP) is a rare inherited disorder caused by ALPL gene mutations, with fracture nonunion being a serious complication. This study investigated the effects of teriparatide and asfotase alfa (AA) on femoral fracture healing of an
Songqi Wang +8 more
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Safety and efficacy of long term asfotase alfa treatment in childhood hypophosphatasia [PDF]
Italian Journal of PediatricsBackground Hypophosphatasia (HPP) is a rare inherited disorder characterized by a deficiency of tissue-non-specific alkaline phosphatase (TNSALP) due to loss-of-function variants of the ALPL gene.
Debora Mariarita d’Angelo +10 more
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Mobility and Quality of Life in Adults with Paediatric-Onset Hypophosphatasia Treated with Asfotase Alfa: Results from UK Managed Access Agreement [PDF]
Advances in TherapyINTRODUCTION: Hypophosphatasia (HPP) is a rare disease caused by deficient tissue-non-specific alkaline phosphatase (ALP) activity. Asfotase alfa is a tissue-non-specific ALP enzyme-replacement therapy which was reimbursed in the UK under a Managed ...
Judith Bubbear +2 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]
Advances in TherapyIntroduction 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 ...
Nick Bishop +2 more
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