Results 51 to 60 of about 172,598 (105)

Hypophosphatasia: A Case of Two Patients With Spinal Cord Compression From Increase in Ligamentous Ossifications During Treatment

, 2021
International audienceTreatment with asfotase alfa has transformed the prognosis of hypophosphatasia in children and improves the bone and muscle signs in adults.
Laroche, Michel, Couture, Guillaume, Valérie Porquet‐Bordes, Guillaume Couture, Salles, Jean Pierre, Michel Laroche, Marie Faruch, Yannick Degboe, Faruch, Marie, Ruyssen-Witrand, Adeline, Porquet-Bordes, Valérie, Adeline Ruyssen‐Witrand, Jean Pierre Salles, Degboe, Yannick   +13 more
core   +1 more source

Profile of asfotase alfa in the treatment of hypophosphatasia: design, development, and place in therapy

, 2018
Sasigarn A Bowden,1 Brian L Foster2 1Division of Endocrinology, Department of Pediatrics, Nationwide Children’s Hospital/The Ohio State University College of Medicine, Columbus, OH 43205, USA; 2Division of Biosciences, College of Dentistry, The ...
Bowden SA, Foster BL
core  

ALFA-1/C9orf72 regulates lipid metabolism through HLH-30/TFEB.

, 2020
(A) Representative images of Nile Red staining in N2, alfa-1(ok3062), HLH-30::GFP-expressing wild-type and HLH-30::GFP-expressing alfa-1(ok3062) mutant C. elegans after 1 day of starvation. Enlarged images of boxed areas are shown in each panel.
Yon Ju Ji (3359057), Tao Zhang (43681), Janet Ugolino (3359054), Jiou Wang (267956), Jiayin Lu (716730), Dohoon Kim (251220)   +5 more
core   +1 more source

Asfotase alfa improved skeletal mineralization and fracture healing in a child with MCAHS. [PDF]

Bone, 2023
Kang M, Wu M, Crane JL.
europepmc   +1 more source

Evolução clínica, radiológica e laboratorial de paciente com hipofosfatasia após dois anos de tratamento com asfotase alfa, com estudo genotípico e fenotípico da família

, 2019
Hypophosphatasia (HPP) is an autosomal dominant or recessive inborn error of metabolism caused by loss of function mutations within the gene that encodes tissuenonspecific alkaline phosphatase (TNSALP), an enzyme with multiple functions including bone ...
Reis, Fernanda Salles [UNIFESP]
core  

The Effect of Asfotase Alfa on Plasma and Urine Pyrophosphate Levels and Pseudofractures in a Patient With Adult-Onset Hypophosphatasia. [PDF]

JBMR Plus, 2023
Hidaka N, Murata H, Tachikawa K, Osaki K, Sekiyama T, Kinoshita Y, Kato H, Hoshino Y, Kimura S, Sunouchi T, Watanabe S, Nangaku M, Makita N, Michigami T, Ito N.   +14 more
europepmc   +1 more source

Pharmacokinetics of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia. [PDF]

J Clin Pharmacol, 2021
Pan WJ, Pradhan R, Pelto R, Seefried L.
europepmc   +1 more source

A Japanese single-center experience of the efficacy and safety of asfotase alfa in pediatric-onset hypophosphatasia. [PDF]

Orphanet J Rare Dis, 2022
Sugiyama Y, Watanabe T, Tajika M, Matsuhashi T, Shimura M, Fushimi T, Ichimoto K, Matsunaga A, Ebihara T, Tsuruoka T, Akiyama T, Murayama K.   +11 more
europepmc   +1 more source

Case Report: Atypical femoral fractures in adult-onset hypophosphatasia treated with low-dose asfotase alfa and adjunctive teriparatide therapy. [PDF]

Front Endocrinol (Lausanne)
Chung YS, Kim KK, S GB, Park JS.
europepmc   +1 more source

Experiences with the use of asfotase alfa in three patients with childhood-onset hypophosphatasia. [PDF]

Endocrinol Diabetes Metab Case Rep
Pigliaru F, Noto D, Brandi ML.
europepmc   +1 more source