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Canavan Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Canavan Disease trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Canavan Disease trials you may qualify forThe main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, i…
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to su…
This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments col…
Canavan Disease is a congenital white matter disorder caused by mutations to the gene encoding for aspartoacylase (ASPA). Expression of ASPA is restricted to ol…
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extr…