Efficacy, safety, and tolerability of chenodeoxycholic acid (CDCA) in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized withdrawal, double-blind, placebo-controlled, crossover phase-3 study
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Published:2025-07
Issue:7
Volume:27
Page:101449
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ISSN:1098-3600
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Container-title:Genetics in Medicine
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language:en
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Short-container-title:Genetics in Medicine
Author:
Kisanuki Yaz Y.ORCID, Nobrega Paulo R., Himes Ryan, Jayadev Suman, Bernat John A., Prakash Vikram, Gibson James B., Larson Austin, Sgobbi Paulo, DeBarber Andrea E., Murphy Edward, Fedor Brian, Wong Po Foo Cheryl, Dutta Rana, Imperiale Michael, Garner Will, Quan Joanne, Vig Pamela, Duell P. Barton, Perez Sarah, Ramdhani Ritesh A., Saute Jonas Alex, Dumitrescu Alina V., Sidhu Alpa, Dubiela Ana Luísa, Dorr Bruno Rutkoski, Martinez Christine, Maciel Franciele Santos, Petrolli Francini, Reyes-Pimentel Gladis, Singh Jaysingh, Nehm Johanna Henriques, Sobreira Neto Manoel Alves, Scherer Mariana Horn, Holida Myrl D., de Castro Pereira Paula, de Lima Serrano Paulo, Braga-Neto Pedro, Aragão Ricardo Evangelista Marrocos de, Kostyk Sandra K., Feddersen Sarah J., Alves Tamires Silva, Czech Theresa M., Rezende Pinto Wladimir Bocca Vieira de
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