Evaluating Personalized (N-of-1) Trials in Rare Diseases: How Much Experimentation Is Enough?

Cheung, K., & Mitsumoto, H. (2022). Evaluating Personalized (N-of-1) Trials in Rare Diseases: How Much Experimentation Is Enough? Harvard Data Science Review. https://doi.org/10.1162/99608f92.e11adff0
Authors:
Ken Cheung
Hiroshi Mitsumoto
Affiliated Authors:
Ken Cheung
Hiroshi Mitsumoto
Columbia Affiliation:
Subjects:
Amyotrophic Lateral Sclerosis and Frontotemporal Dementia (OpenAlex Topic)
Gene Therapy for Spinal Muscular Atrophy (OpenAlex Topic)
Prion Diseases: Causes and Molecular Basis (OpenAlex Topic)
Author Keywords:
als
heterogeneity of treatment effects (hte)
minimally clinically important heterogeneity
patient-centered research
rare diseases
sample size formulae
elink
Publication Type:
Article
Unique ID:
10.1162/99608f92.e11adff0
PMID:
38283317
DOI:
10.1162/99608f92.e11adff0
Journal:
Harvard Data Science Review
Publication Date:
Data Source:
OpenAlex
Source Link:
View OpenAlex Record
View PubMed Record

Record Created: