@article{6e4244bdf1ee498f865778f678d0dc1c,
title = "Designing clinical trials for rare diseases: unique challenges and opportunities",
author = "Chiara Pizzamiglio and Vernon, \{Hilary J.\} and Hanna, \{Michael G.\} and Pitceathly, \{Robert D.S.\}",
note = "Funding Information: C.P. is supported by a Clore Duffield Foundation grant. R.D.S.P. is supported by a Medical Research Council (UK) Clinician Scientist Fellowship (MR/S002065/1). R.D.S.P. and M.G.H. are funded by a Medical Research Council (UK) strategic award to establish an International Centre for Genomic Medicine in Neuromuscular Diseases (ICGNMD) (MR/S005021/1). ",
year = "2022",
month = dec,
doi = "10.1038/s43586-022-00100-2",
language = "English (US)",
volume = "2",
journal = "Nature Reviews Methods Primers",
issn = "2662-8449",
publisher = "Springer Nature",
number = "1",
}