View clinical trials related to Vanishing White Matter Disease.
Filter by:ABBV-CLS-7262 is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult and pediatric subjects. This is a 96-week, open-label, multiple cohort study enrolling adults and pediatric subjects with Vanishing White Matter disease who are 6 years or older. Subjects will attend regular visits during the course of the study and complete medical assessments, blood tests, checking for side effects, and completing questionnaires.
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.