Genetics in Medicine Open (Jan 2024)
P142: Efficacy, safety and tolerability of chenodeoxycholic acid in adult patients with cerebrotendinous xanthomatosis (RESTORE): A randomized, placebo-controlled phase 3 study*
- Andrea DeBarber,
- Yaz Kisanuki,
- Paulo Nobrega,
- Ryan Himes,
- Suman Jayadev,
- John Bernat,
- Vikram Prakash,
- James Gibson,
- Austin Larson,
- Paulo Sgobbi,
- Edward Murphy,
- Brian Fedor,
- Cheryl Wong Po Foo,
- Rana Dutta,
- Michael Imperiale,
- Jolan Terner-Rosenthal,
- Will Garner,
- Pamela Vig,
- P. Barton Duell,
- Sarah Perez,
- Ritesh Ramdhani,
- Jonas Saute
Affiliations
- Andrea DeBarber
- Oregon Health and Science University, Portland, OR
- Yaz Kisanuki
- The Ohio State University Wexner Medical Center, Columbus, OH
- Paulo Nobrega
- Hospital Universitario Walter Cantidio – Universidade Federal do Ceara, Brazil
- Ryan Himes
- Ochsner Medical Center, New Orleans, LA
- Suman Jayadev
- University of Washington, Seattle, WA
- John Bernat
- University of Iowa Hospitals and Clinics, Iowa City, IA
- Vikram Prakash
- Arnold Palmer Children’s Hospital and Orlando Health, Orlando, FL
- James Gibson
- Dell Children’s Medical Center, Austin, TX
- Austin Larson
- Children’s Hospital Colorado, University of Colorado School of Medicine, Aurora, CO
- Paulo Sgobbi
- Universidade Federal de Sao Paulo – Escola Paulista de Medicine, Brazil
- Edward Murphy
- Travere Therapeutics, San Diego, CA
- Brian Fedor
- Travere Therapeutics, San Diego, CA
- Cheryl Wong Po Foo
- Travere Therapeutics, San Diego, CA
- Rana Dutta
- Mirum Pharmaceuticals, Inc., Foster City, CA
- Michael Imperiale
- Mirum Pharmaceuticals, Inc., Foster City, CA
- Jolan Terner-Rosenthal
- Mirum Pharmaceuticals, Inc., Foster City, CA
- Will Garner
- Mirum Pharmaceuticals, Inc., Foster City, CA
- Pamela Vig
- Mirum Pharmaceuticals, Inc., Foster City, CA
- P. Barton Duell
- Oregon Health and Science University, Portland, OR
- Sarah Perez
- Tulane University School of Medicine, New Orleans, LA
- Ritesh Ramdhani
- Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY
- Jonas Saute
- Medical Genetics Service, Hospital de Clinicas de Porto Alegre, Brazil
- Journal volume & issue
-
Vol. 2
p. 101039
