Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis
2020; Massachusetts Medical Society; Volume: 383; Issue: 10 Linguagem: Inglês
10.1056/nejmoa1916945
ISSN1533-4406
AutoresSabrina Paganoni, Eric A. Macklin, Suzanne Hendrix, James Berry, Michael A. Elliott, Samuel Maiser, Chafic Karam, James B. Caress, Margaret Owegi, Adam Quick, James Wymer, Stephen A. Goutman, Daragh Heitzman, Terry Heiman‐Patterson, Carlayne E. Jackson, Colin Quinn, Jeffrey D. Rothstein, Edward J. Kasarskis, Jonathan Katz, Liberty Jenkins, Shafeeq Ladha, Jonathan Mill, Stephen N. Scelsa, Tuan Vu, Christina N. Fournier, Jonathan D. Glass, Kristin Johnson, Andrea Swenson, Namita Goyal, Gary L. Pattee, Patricia L. Andres, Suma Babu, Marianne Chase, Derek D’Agostino, Samuel P. Dickson, Noel Ellison, Meghan Hall, Kent Hendrix, Gale Kittle, Michelle McGovern, Joseph Ostrow, Lindsay Pothier, Rebecca Randall, Jeremy M. Shefner, Alexander Sherman, Eric Tustison, Prasha Vigneswaran, Jason Walker, Hong Yu, James Chan, Janet Wittes, Joshua Cohen, Justin Klee, Kent Leslie, Rudolph E. Tanzi, Walter Gilbert, Patrick Yeramian, David Schoenfeld, Merit Cudkowicz,
Tópico(s)Prion Diseases and Protein Misfolding
ResumoSodium 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.
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