Hypophosphatasia: presentation and response to asfotase alfa.

Farah Alsarraf Dalal S AliK AlmonaeiHatim Al-Alwani Aliya A Khan Maria Luisa Brandi

Published in: Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA (2023)

HPP treatment with AA in individuals with both pediatric and adult-onset forms resulted in significant subjective improvement in musculoskeletal and cognitive manifestations in addition to patients' quality of life. The drug was well tolerated in 6 patients. 1 patient discontinued therapy because of minor adverse effects with myalgias.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
prognostic factors
peritoneal dialysis
emergency department
stem cells
patient reported outcomes
physical activity
childhood cancer