- Title: Subtitle
- P142: Efficacy, safety and tolerability of chenodeoxycholic acid in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized, placebo-controlled phase 3 study
- Creators
- Andrea DeBarber - University of PortlandYaz Kisanuki - The Ohio State University Wexner Medical CenterPaulo Nobrega - Hospital Universitário Walter CantídioRyan Himes - Ochsner Medical CenterSuman Jayadev - University of WashingtonJohn Bernat - University of IowaVikram Prakash - Orlando HealthJames Gibson - Dell Children's Medical Center of Central TexasAustin Larson - Children's Hospital ColoradoPaulo Sgobbi - Universidade Federal de São PauloEdward Murphy - Travere Therapeutics, San Diego, CABrian Fedor - Travere Therapeutics, San Diego, CACheryl Wong Po Foo - Travere Therapeutics, San Diego, CARana Dutta - Mirum Pharmaceuticals, Inc., Foster City, CAMichael Imperiale - Mirum Pharmaceuticals, Inc., Foster City, CAJolan Terner-Rosenthal - Mirum Pharmaceuticals, Inc., Foster City, CAWill Garner - Mirum Pharmaceuticals, Inc., Foster City, CAPamela Vig - Mirum Pharmaceuticals, Inc., Foster City, CAP. Barton Duell - University of PortlandSarah Perez - Tulane UniversityRitesh Ramdhani - Donald & Barbara Zucker School of Medicine at Hofstra/NorthwellJonas Saute - Hospital de Clínicas de Porto Alegre
- Resource Type
- Abstract
- Publication Details
- Genetics in Medicine Open, Vol.2(Suppl. 1), 101039
- DOI
- 10.1016/j.gimo.2024.101039
- ISSN
- 2949-7744
- Publisher
- Elsevier Inc
- Language
- English
- Date published
- 2024
- Academic Unit
- Stead Family Department of Pediatrics; Medical Genetics and Genomics
- Record Identifier
- 9984562589002771
Abstract
P142: Efficacy, safety and tolerability of chenodeoxycholic acid in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized, placebo-controlled phase 3 study
Genetics in Medicine Open, Vol.2(Suppl. 1), 101039
2024
DOI: 10.1016/j.gimo.2024.101039
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