Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease
Condition: Wilson Disease Interventions: Genetic: UX701; Other: Placebo; Drug: oral corticosteroids; Drug: Placebo for oral corticosteroids Sponsor: Ultragenyx Pharmaceutical Inc Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - May 13, 2021 Category: Research Source Type: clinical trials
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Condition: Wilson Disease Intervention: Drug: ALXN1840 Sponsor: Alexion Pharmaceuticals Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - October 2, 2020 Category: Research Source Type: clinical trials