Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Paganoni, Sabrina; A. Macklin, Eric; Hendrix, Suzanne; D. Berry, James; A. Elliott, Michael; Maiser, Samuel; Karam, Chafic; B. Caress, James; Owegi, Margaret; Quick, Adam; Wymer, James; A. Goutman, Stephen; Heitzman, Daragh; Heiman‐Patterson, Terry; E. Jackson, Carlayne; Quinn, Colin; D. Rothstein, Jeffrey; J. Kasarskis, Edward; Katz, Jonathan; Jenkins, Liberty; Ladha, Shafeeq; Mill, Jonathan; N. Scelsa, Stephen; Vu, Tuan; Fournier, Christina; D. Glass, Jonathan; Johnson, Kristin; Swenson, Andrea; Goyal, Namita; L. Pattee, Gary; L. Andres, Patricia; Babu, Suma; Chase, Marianne; D’Agostino, Derek; P. Dickson, Samuel; Ellison, Noel; Hall, Meghan; Hendrix, Kent; Kittle, Gale; McGovern, Michelle; Ostrow, Joseph; Pothier, Lindsay; Randall, Rebecca; M. Shefner, Jeremy; Sherman, Alexander; Tustison, Eric; Vigneswaran, Prasha; Walker, Jason; Yu, Hong; Chan, James; Wittes, Janet; Cohen, Joshua; Klee, Justin; Leslie, Kent; E. Tanzi, Rudolph; Gilbert, Walter; Yeramian, Patrick; Schoenfeld, David; Cudkowicz, Merit

doi:10.1056/nejmoa1916945

Public

Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Published in New England Journal of Medicine • Sep 2, 2020

Authors:

Sabrina Paganoni

Eric A. Macklin

Suzanne Hendrix

Abstract

BACKGROUND: Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy and safety of a combination of the two compounds in persons with amyotrophic lateral sclerosis (ALS) are not known. METHODS: In this multicenter, randomized, double-blind ...

Subject

Phenylbutyrate

Amyotrophic lateral sclerosis

Medicine

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