Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies-Reference-Cited by-同舟云学术

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Published:2012 Issue:1 Volume:7 Page:91
ISSN:1750-1172
Container-title:Orphanet Journal of Rare Diseases
language:en
Short-container-title:Orphanet Journal of Rare Diseases

Author:

Germain Dominique P,Giugliani Roberto,Hughes Derralynn A,Mehta Atul,Nicholls Kathy,Barisoni Laura,Jennette Charles J,Bragat Alexander,Castelli Jeff,Sitaraman Sheela,Lockhart David J,Boudes Pol F

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Genetics(clinical),General Medicine

Reference22 articles.

1. Fabry disease

2. Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease

3. Enzyme Replacement Therapy in Fabry Disease

4. Accelerated transport and maturation of lysosomal α–galactosidase A in Fabry lymphoblasts by an enzyme inhibitor

5. Mutant α-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin

Cited by 103 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Fabry nephropathy: a treatable cause of chronic kidney disease;Rare Disease and Orphan Drugs Journal;2024-07-11

2. Neuropathy and pain in Fabry disease;Rare Disease and Orphan Drugs Journal;2024-07-08

3. A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry’s Disease;Expert Opinion on Pharmacotherapy;2024-04-12

4. Carbohydrate‐Based Therapeutics for Lysosomal Storage Disorders;Carbohydrate‐Based Therapeutics;2023-09-05

5. Impact of kidney biopsy on deciding when to initiate enzyme replacement therapy in children with Fabry disease;Pediatric Nephrology;2023-07-20