Cargando…
Randomized and non-randomized designs for causal inference with longitudinal data in rare disorders
In the United States, approximately 7000 rare diseases affect 30 million patients, and only 10% of these diseases have existing therapies. Sound study design and causal inference methods are essential to demonstrate the therapeutic efficacy, safety, and effectiveness of new therapies. In the rare di...
| Autores principales: | , |
|---|---|
| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2021
|
| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8609847/ https://www.ncbi.nlm.nih.gov/pubmed/34814939 http://dx.doi.org/10.1186/s13023-021-02124-5 |