Effect of Asfotase Alfa in the Treatment of Hypophosphatasia- A Systematic Review. [PDF]
Hypophosphatasia (HPP) is a life-threatening disease that occurs due to the mutation of the TNSALP (Tissue nonspecific isoenzyme of alkaline phosphatase) encoding gene. There is no approved treatment for Hypophosphatasia.
Jaswanthi N +7 more
europepmc +2 more sources
Altered Thyroid Function Tests Observed in Hypophosphatasia Patients Treated with Asfotase Alfa. [PDF]
Background. Asfotase alfa is the only approved treatment that can normalize mineralization in patients with hypophosphatasia (HPP). Its interference in alkaline phosphatase (ALP) dependent immunoassays has been reported. Objective.
Kato H +6 more
europepmc +2 more sources
Bone turnover and mineral metabolism in adult patients with hypophosphatasia treated with asfotase alfa. [PDF]
Summary There is limited understanding of how asfotase alfa affects mineral metabolism and bone turnover in adults with pediatric-onset hypophosphatasia.
Seefried L, Rak D, Petryk A, Genest F.
europepmc +2 more sources
Excellent response to asfotase alfa treatment in an adolescent patient with hypophosphatasia. [PDF]
Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by deficient activity of alkaline phosphatase, causing defective mineralization of bones and teeth.
Strandbech OS, Lund A, Ostergaard E.
europepmc +2 more sources
Effectiveness and safety of asfotase alfa for people with hypophosphatasia: a plain language summary of three studies. [PDF]
What 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.
Jasmi FA, Belaya Z.
europepmc +2 more sources
ADULT-ONSET HYPOPHOSPHATASIA: BEFORE AND AFTER TREATMENT WITH ASFOTASE ALFA. [PDF]
Objective: To review the diagnosis and clinical course of a woman with hypophosphatasia who is being treated with newly approved enzyme replacement therapy, asfotase alfa. Method: Clinical and laboratory data are presented.
Magdaleno AL +4 more
europepmc +3 more sources
Effects of asfotase alfa on fracture healing of adult patient with hypophosphatasia and literature review. [PDF]
Objective 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
Wang S +8 more
europepmc +2 more sources
Efficacy of asfotase alfa in a patient with adult-onset hypophosphatasia without obvious bone lesions: a case report with review of literature. [PDF]
The use of asfotase alfa, a bone-targeted recombinant alkaline phosphatase (ALP) enzyme, for the treatment of adult-onset hypophosphatasia (HPP) remains controversial, particularly in patients without evident bone abnormalities.
Nishikage S +11 more
europepmc +2 more sources
Physical Function and Health-Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric-Onset Hypophosphatasia. [PDF]
Hypophosphatasia (HPP) is a rare, inherited, metabolic disease characterized by tissue‐nonspecific alkaline phosphatase deficiency resulting in musculoskeletal and systemic clinical manifestations.
Genest F, Rak D, Petryk A, Seefried L.
europepmc +2 more sources
Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry. [PDF]
Background 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 ...
Kishnani PS +10 more
europepmc +2 more sources

