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

• Main Authors: Ariana P. Mullin (Author), Diane Corey (Author), Emily C. Turner (Author), Richard Liwski (Author), Daniel Olson (Author), Jackson Burton (Author), Sudhir Sivakumaran (Author), Lynn D. Hudson (Author), Klaus Romero (Author), Diane T. Stephenson (Author), Jane Larkindale (Author)
• Format: Book
• Published: Wiley, 2021-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Interest in drug development for rare diseases has expanded dramatically since the Orphan Drug Act was passed in 1983, with 40% of new drug approvals in 2019 targeting orphan indications. However, limited quantitative understanding of natural history and disease progression hinders progress and incr...