Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease-Reference-Cited by-同舟云学术

Standardized Data Structures in Rare Diseases: CDISC User Guides for Duchenne Muscular Dystrophy and Huntington’s Disease

Published:2020-08-25 Issue:1 Volume:14 Page:214-221
ISSN:1752-8054
Container-title:Clinical and Translational Science
language:en
Short-container-title:Clin Transl Sci

Author:

Mullin Ariana P.¹,Corey Diane¹,Turner Emily C.¹,Liwski Richard¹,Olson Daniel¹,Burton Jackson¹,Sivakumaran Sudhir¹,Hudson Lynn D.¹,Romero Klaus¹,Stephenson Diane T.¹,Larkindale Jane¹

Affiliation:

1. Critical Path Institute Tucson Arizona USA

Funder

Parent Project Muscular Dystrophy

CHDI Foundation

Food and Drug Administration

Publisher

Wiley

Subject

General Pharmacology, Toxicology and Pharmaceutics,General Biochemistry, Genetics and Molecular Biology,General Medicine,General Neuroscience

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1111/cts.12845

Reference32 articles.

1. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study

2. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

3. Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

4. A post-hoc subgroup analysis of outcomes in the first phase III clinical study of edaravone (MCI-186) in amyotrophic lateral sclerosis

5. Regulatory-accepted drug development tools are needed to accelerate innovative CNS disease treatments

Cited by 8 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. How to customize common data models for rare diseases: an OMOP-based implementation and lessons learned;Orphanet Journal of Rare Diseases;2024-08-14

2. Fine-tuning large language models for rare disease concept normalization;Journal of the American Medical Informatics Association;2024-06-03

3. Scoping review of the recommendations and guidance for improving the quality of rare disease registries;Orphanet Journal of Rare Diseases;2024-05-06

4. Next-generation therapeutics for rare genetic disorders;Mutagenesis;2024-02-09

5. NCI-EVS: Building the Semantic Infrastructure to Support CDISC Data Standards and Real-World Data;Journal of the Society for Clinical Data Management;2023-06-19