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Zellweger Spectrum
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Zellweger Spectrum trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Zellweger Spectrum 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…
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in p…
A series of N-of-1, crossover, randomized, placebo-controlled, double-blinded trial. Hydroxychloroquine (HCQ) and a crossover to placebo (order is randomized an…
The purpose of this study is to characterize the symptoms of Zellweger Spectrum Disorder (ZSD) and related peroxisome disorders, and to assess the quality of li…