A framework for applying unfamiliar trial designs in studies of rare diseases-Reference-Cited by-同舟云学术

A framework for applying unfamiliar trial designs in studies of rare diseases

Published:2011-10 Issue:10 Volume:64 Page:1085-1094
ISSN:0895-4356
Container-title:Journal of Clinical Epidemiology
language:en
Short-container-title:Journal of Clinical Epidemiology

Author:

Gupta Samir,Faughnan Marie E.,Tomlinson George A.,Bayoumi Ahmed M.

Publisher

Elsevier BV

Subject

Epidemiology

Reference55 articles.

1. EURORDIS: European Organisation for Rare Diseases. Rare diseases: understanding this Public Health Priority. 2005. Available at http://www.eurordis.org/IMG/pdf/princeps_document-EN.pdf. Accessed August 10, 2009.

2. Orphan drugs: legal aspects, current situation;Lavandeira;Haemophilia,2002

3. Why rare diseases are an important medical and social issue;Schieppati;Lancet,2008

4. Adopting orphan drugs—two dozen years of treating rare diseases;Haffner;N Engl J Med,2006

5. Adaptive design in clinical research: issues, opportunities, and recommendations;Chang;J Biopharm Stat,2006

Cited by 82 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Cannabidiol (Epidyolex®) for severe behavioral manifestations in patients with tuberous sclerosis complex, mucopolysaccharidosis type III and fragile X syndrome: protocol for a series of randomized, placebo-controlled N-of-1 trials;BMC Psychiatry;2024-01-04

2. Effectiveness of L-serine supplementation in children with a GRIN2B loss-of-function mutation: Rationale and protocol for single patient (n-of-1) multiple cross-over trials;Contemporary Clinical Trials Communications;2023-12

3. Toward responsible clinical n-of-1 strategies for rare diseases;Drug Discovery Today;2023-10

4. Randomized controlled trial data for successful new drug application for rare diseases in the United States;Orphanet Journal of Rare Diseases;2023-04-19

5. Feasibility of a Platform Trial Design for the Development of Mobile Health Applications: A Review;Telemedicine and e-Health;2023-04-01