Hypophosphatasia: presentation and response to asfotase alfa

ConclusionHPP 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.
Source: Osteoporosis International - Category: Orthopaedics Source Type: research