官方服务:
资源简介:
FMD, AIX treatment agalsidase beta vs placebo in Fabry patients
应用场景:
创建时间:
2020-08-03
相关数据集
Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy
Background Approximately 13% of boys with Duchenne muscular dystrophy (DMD) have a nonsense mutation in the dystrophin gene, resulting in a premature stop codon in the corresponding mRNA and failure t
NIAID Data Ecosystem50
Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial
The study objective was to assess the effect of vutrisiran, an RNA interference therapeutic that reduces transthyretin (TTR) production, in patients with hereditary transthyretin (ATTRv) amyloidosis w
Mendeley Data2024-06-27 更新30
dataset Fabry disease
FMD, AIX treatment agalsidase beta vs placebo in Fabry patients
Figshare2020-08-03 更新10
A real-world pharmacovigilance analysis for agalsidase beta: findings from the FDA adverse event reporting database
Fabry disease (FD), an X-linked lysosomal disorder, is marked by a lack of alpha-galactosidase A (α-Gal A). Agalsidase beta, a recombinant form of α-Gal A, is fundamental to enzyme replacement therapy
Figshare2024-12-21 更新30
Global Fabry Disease Treatment Market 2025 To 2034
Fabry Disease Treatment Market Size, Trends and Insights By Treatment (Enzyme Replacement Therapy (ERT), Chaperone Treatment, Substrate Reduction Therapy (SRT), Others), By Route of Administration (In
NIAID Data Ecosystem20



