Hypophosphatasia: presentation and response to asfotase alfa.

Autor: Alsarraf, F., Ali, D.S., Almonaei, K., Al-Alwani, H., Khan, A.A., Brandi, M.L.

Publikováno v: Osteoporosis International; Apr2024, Vol. 35 Issue 4, p717-725, 9p

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Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia.

Autor: Ali DS; Division of Endocrinology and Metabolism, McMaster University., Mirza RD; Department of Health Research Methods, Evidence, and Impact, McMaster University., Alsarraf F; Division of Endocrinology and Metabolism, McMaster University., Hussein S; Division of Endocrinology and Metabolism, McMaster University., Abu Alrob H; Department of Health Research Methods, Evidence, and Impact, McMaster University., Appelman-Dijkstra NM; Department of Internal Medicine, Division of Endocrinology, Center for Bone Quality, Leiden University Medical Center, Leiden, the Netherlands., Beck-Nielsen SS; Centre for Rare Diseases, Aarhus University Hospital, Aarhus, Denmark.; Department of Clinical Medicine, Aarhus University, Aarhus, Denmark., Biosse-Duplan M; Faculty of Dentistry, Université Paris Cité, Paris, France; Institut Imagine, INSERM 1163, Paris, France; APHP, Paris, France., Brandi ML; Vita-Salute San Raffaele University, Milan, Italy., Carpenter TO; Departments of Pediatrics (Endocrinology), and Orthopedics and Rehabilitation, Yale University School of Medicine, New Haven, CT, USA., Chaussain C; Faculty of Dentistry, Université Paris Cité and APHP, Paris, France., Cohen-Solal M; Department of Rheumatology and Reference Center for Rare Bone Diseases, hopital Lariboisiere, Paris, France., Crowley RK; Rare Disease Clinical Trial Network, University College Dublin, Ireland., Dandurand K; Université de Sherbrooke, Sherbrooke, Québec, Canada., Florenzano P; Department of Endocrinology, School of Medicine Pontificia Universidad Católica de Chile., Fukumoto S; Tamaki-Aozora Hospital, Japan., Gagnon C; Department of Medicine, Université Laval, Quebec City, Canada; CHU de Québec-Université Laval Research Centre, Quebec City, Canada., Goodyer P; Research Institute of the McGill University Health Centre, Montreal, Canada., Grasemann C; Department of Pediatrics, Division of Rare Diseases, Katholisches Klinikum Bochum and Ruhr-University Bochum, Bochum, Germany., Imel EA; Department of Medicine and Pediatrics, Indiana University School of Medicine., Jan de Beur SM; University of Virginia School of Medicine, Charlottesville, VA, USA., Lehman A; University of British Columbia; Adult Metabolic Diseases Clinic, Vancouver, BC, Canada., Lewiecki EM; New Mexico Clinical Research & Osteoporosis Center, Albuquerque, NM, USA., Morgante E; University of Waterloo, Waterloo, ON, Canada., Ward LM; Children's Hospital of Eastern Ontario, Department of Pediatrics, University of Ottawa, Ottawa, Ontario, Canada., Khan AA; Division of Endocrinology and Metabolism, McMaster University., Guyatt G; Department of Health Research Methods, Evidence, and Impact, McMaster University.; Department of Medicine, McMaster University, Hamilton, Ontario, Canada.; MAGIC Evidence Ecosystem Foundation [ www.magicevidence.org ], Lovisenberggata 17C, 0456 Oslo, Norway.

Publikováno v: The Journal of clinical endocrinology and metabolism [J Clin Endocrinol Metab] 2024 Dec 23. Date of Electronic Publication: 2024 Dec 23.