A checklist for clinical trials in rare disease

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This paper, titled 'A checklist for clinical trials in rare disease: obstacles and anticipatory actions - lessons learned from the FOR-DMD trial', outlines the challenges and lessons learned from the FOR-DMD trial about running trials in the rare disease space. 

A checklist for clinical trials in rare disease: obstacles and anticipatory actions - lessons learned from the FOR-DMD trial. - Rebecca A. Crow et. al. 

Read the paper here