Results 121 to 130 of about 714 (150)

Hypophosphatasia: presentation and response to asfotase alfa

Osteoporosis International, 2023
Hypophosphatasia (HPP) is a rare bone disease with limited scientific evidence on the tolerability and safety of its novel treatment, Asfotase Alfa (AA). We report 7 HPP patients' heterogenous presentations and the significant improvement in various clinical outcomes attained with AA shedding light on this highly effective and safe therapy ...
Farah Alsarraf, Dalal S Ali, Hatim Al-Alwani   +2 more
exaly   +3 more sources

Dental outcomes for children receiving asfotase alfa for hypophosphatasia

Bone, 2021
Hypophosphatasia, a genetic disease impeding development of teeth and bones, is associated with premature exfoliation of primary teeth. Hypophosphatasia is caused by mutations in the ALPL gene, which encodes the tissue non-specific form of alkaline phosphatase.
Robert J Schroth, VÍCTOR H K Lee, Cheryl Rockman-Greenberg   +2 more
exaly   +3 more sources

Status Epilepticus due to Asfotase Alfa Interruption in Perinatal Severe Hypophosphatasia

Pediatric Neurology, 2022
Hypophosphatasia (HPP), an inherited, metabolic disorder caused by loss-of-function mutations in the ALPL gene, affects not only bone and tooth mineralization but also central nervous system (CNS) function, resulting in vitamin B6/pyridoxine-responsive seizures. Asfotase alfa treatment mainly improves the skeletal manifestations of HPP.
Eri Ogawa, Kazuhiro Shimura, Hiroshi Yoshihashi   +2 more
exaly   +3 more sources

Efficacy and safety of asfotase alfa in patients with hypophosphatasia: A systematic review

Bone
Hypophosphatasia (HPP) is a rare genetic disorder characterized by defective bone mineralization, leading to skeletal abnormalities and systemic complications. Asfotase alfa, a recombinant human tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy, has emerged as a promising treatment for HPP.
Amirhossein Ghaseminejad-Raeini, Yasaman Tavakoli, Alireza Azarboo   +2 more
exaly   +3 more sources

Asfotase-alfa

Reactions Weekly, 2017
core   +4 more sources

Asfotase Alfa hypersensitivity: an outpatient 8-steps desensitization protocol

Immunologic Research, 2021
Scudu S., Ghisu A., Costanzo G., Barca M. P., Firinu D., Del Giacco S.   +5 more
openaire   +4 more sources

Asfotase-alfa

Reactions Weekly
core   +3 more sources

Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial [PDF]

Lancet Diabetes and Endocrinology,the, 2019
BACKGROUND: Our previous phase 2, open-label study of 11 infants and young children with life-threatening perinatal or infantile hypophosphatasia showed 1 year safety and efficacy of asfotase alfa, an enzyme replacement therapy.
Michael P Whyte, Jill H Simmons, Scott Moseley   +2 more
exaly   +2 more sources

Asfotase Alfa: A Review in Paediatric-Onset Hypophosphatasia

Drugs, 2016
Hypophosphatasia (HPP) is a rare inheritable disease that results from loss-of-function mutations in the ALPL gene encoding tissue-nonspecific alkaline phosphatase (TNSALP). Therapeutic options for treating the underlying pathophysiology of the disease have been lacking, with the mainstay of treatment being management of symptoms and supportive care ...
openaire   +2 more sources

Asfotase alfa: Enzyme replacement for the treatment of bone disease in hypophosphatasia

Drugs of Today, 2016
Hypophosphatasia (HPP) is a rare disease caused by loss-of-function mutations in the tissue-nonspecific alkaline phosphatase (TNAP, TNSALP) gene. HPP causes a multisystemic syndrome with a predominant bone phenotype. The clinical spectrum ranges from high lethality in early onset (
C, Hofmann, L, Seefried, F, Jakob
openaire   +2 more sources