A plea to provide best evidence in trials under sample-size restrictions: the example of pioglitazone to resolve leukoplakia and erythroplakia in Fanconi anemia patients

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Research methods to change clinical practice for patients with rare cancers

(2016) Lucinda Billingham et al.   LANCET ONCOLOGY

Abstract 914: Pioglitazone and metformin as potential chemopreventative treatments in Fanconi anemia related oral squamous carcinoma

(2015) Kim Miller et al.   CANCER RESEARCH

The role of nonrandomized trials in the evaluation of oncology drugs

(2015) R Simon et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Do we need randomised trials for rare cancers?

(2015) Vinay Prasad et al.   EUROPEAN JOURNAL OF CANCER

A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov

(2014) Stuart A Bell et al.   Orphanet Journal of Rare Diseases

Clinical Trials in Rare Disease

(2013) Erika F. Augustine et al.   JOURNAL OF CHILD NEUROLOGY

Rare Cancer Trial Design: Lessons from FDA Approvals

(2012) H. Gaddipati et al.   CLINICAL CANCER RESEARCH

Clinical research for rare disease: Opportunities, challenges, and solutions

(2008) Robert C. Griggs et al.   MOLECULAR GENETICS AND METABOLISM