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Canavan Disease
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Canavan Disease trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
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The 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…