The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.
The purpose of this grant is to address and fill the gaps in clinical trial readiness for rare neurological and neuromuscular conditions which are on the brink of having candidate therapeutics tested in clinical trials but face challenges due to the absence of validated clinical outcome assessment measures or biomarkers. This may hinder the success of such trials. The grant aims to support multi-site, prospective clinical projects that tackle the essential requirements for clinical trial readiness, focusing on the establishment of clinically valid biomarkers and clinical outcome assessment measures specifically designed for use in forthcoming trials. The National Institute of Neurological Disorders and Stroke (NINDS) and the National Center for Advancing Translational Sciences (NCATS) aim to enhance the quality and success rate of future clinical trials in these fields by backing studies that lead to the readiness of trials.