2024
WED-164 Interim safety results of the ongoing international phase I/II GATEWAY gene therapy trial with VTX-801 conducted in adult patients with Wilson disease
Sandahl T, Lee W, Ala A, Gonzalez-Peralta R, Medici V, Askari F, Rudnick S, Lauer U, Schmidt H, Valero S, Gonzalez G, Combal J, D'Antiga L, Benichou B, Schilsky M. WED-164 Interim safety results of the ongoing international phase I/II GATEWAY gene therapy trial with VTX-801 conducted in adult patients with Wilson disease. Journal Of Hepatology 2024, 80: s714-s715. DOI: 10.1016/s0168-8278(24)02021-x.Peer-Reviewed Original Research
2023
P20 Patterns of ALT, AST and 24-hour urine copper in adult patients with treated Wilson disease: results from an international multi-site registry at enrolment and over the course of 3 years
Camarata M, To U, Coskun A, Maciejewski K, Embel V, Ayhan E, Gonzalez-Peralta R, Ala A, Schilsky M. P20 Patterns of ALT, AST and 24-hour urine copper in adult patients with treated Wilson disease: results from an international multi-site registry at enrolment and over the course of 3 years. 2023, a25.1-a25. DOI: 10.1136/gutjnl-2023-basl.36.Peer-Reviewed Original Research
2020
P12 Effect of liver disease, neurological disease and mental health issues on quality of life in patients with wilson disease
Camarata M, Ala A, Maciejewski K, To U, Zimbrean P, Rubman S, Coskun A, Patel A, Wadhwa A, Apdik T, Tomlin R, Deng Y, Gonzalez-Peralta R, Schilsky M. P12 Effect of liver disease, neurological disease and mental health issues on quality of life in patients with wilson disease. Gut 2020, 69: a13-a13. DOI: 10.1136/gutjnl-2020-basl.23.Peer-Reviewed Original ResearchMental health issuesWilson's diseaseHealth issuesSeverity of liverMental health QOLPhysical health QoLReview of imagingP-QOL scoresQuality of lifeFIB4 scoreAdult patientsRegistry studyNeurological assessmentPhysical health issuesChronic diseasesOutcome measuresPatientsNeurological diseasesWD patientsQoLAssess qualityDiseaseCirrhosisSignificant differencesScores