Back to search
Zellweger Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Zellweger Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Zellweger Syndrome trials you may qualify forThe goal of this observational study is to define the course of the retinal degeneration in a ZSD patient cohort. The objective of this study is to gather info…
The Peroxisome Biogenesis Disorders (PBD) are a group of inherited disorders due to defects in peroxisome assembly causing complex developmental and metabolic s…
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to su…
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-rel…
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extr…
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bil…