Clinical Trials in Rare Diseases - General Resources

Batten Disease Resources and Links

Here we provide some general resources relevant to clinical trials in rare diseases, including a list of relevant articles and website links. Please note that we cannot directly post published articles, due to copyright laws. However, wherever available, we have provided a link to a free, downloadable version of the article. Please contact us if you wish to suggest other materials to be shared here.

• Basic Questions and Answers about Clinical Trials
• National Institutes of Health: Batten disease information page
• NIH Clinical Trials and You
• General Resources (Articles, Books, links, etc.)
• FDA Qualification of CLINICAL OUTCOME ASSESSMENTS (COAs)
• FDA Roadmap to PATIENT-FOCUSED OUTCOME MEASUREMENT in Clinical Trials
• Understanding Clinical Trial Design: A Tutorial for Research Advocates
• Rare Disease Task Force Report on patient registries in the field of rare diseases

Support for this conference was provided by:

The National Institutes of Health – National Institute of Neurological Diseases and Stroke (NIH/NINDS): 1R13 NS078981, Outcomes and Infrastructure for Phase III Clinical Trials in JNCL - Batten Disease

The University of Rochester Child Neurology Research Fund