Abstract
The treatment of rare disorders is often driven by experience and expert opinion rather than formal clinical trials because of the significant barriers in place to conducting traditional random, placebo controlled, national studies. Dr. Doug Kerr graphically illustrates these problems in the treatment of inborn errors of mitochondrial oxidative phosphorylation as reviewed elsewhere in this issue. New models and additional funding for studying the ultra rare diseases that are the purview of metabolic medicine are desperately needed to move the field forward in an evidence-based fashion. In this commentary, the challenges associated with conducting clinical trials in ultra-rare disorders are discussed and potential solutions suggested.
Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
