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Peroxisome Biogenesis Disorder
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Peroxisome Biogenesis Disorder trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Peroxisome Biogenesis Disorder trials may be worth asking aboutNorth America
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The 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…