1. Small Clinical Trials: Issues and Challenges,2001
2. Clinical trials and rare diseases;Lagakos;N Engl J Med,2003
3. Rare diseases and the assessment of intervention: what sorts of clinical trials can we use?;Wilcken;J Inherit,2001
4. Challenges in the design and conduct of therapeutic trials in channel disorders;Venance;Neurotherapeutics,2007
5. Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics, U.S. Department of Health and Human Services Food and Drug Administration, Center for Drug Evaluation and Research (CDER) Center for Biologics Evaluation and Research (CBER), February 2010.