Trial of Sodium Phenylbutyrate–Taurursodiol for Amyotrophic Lateral Sclerosis

Sodium phenylbutyrate combined with taurursodiol reduces neuronal endoplasmic reticulum stress and mitochondrial dysfunction in experimental models. In a randomized trial, the combination slowed the rate of progression of ALS but did not affect the slow vital capacity or isometric muscle strength.

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Veröffentlicht in:	The New England journal of medicine 2020-09, Vol.383 (10), p.919-930
Hauptverfasser:	Paganoni, Sabrina, Macklin, Eric A, Hendrix, Suzanne, Berry, James D, Elliott, Michael A, Maiser, Samuel, Karam, Chafic, Caress, James B, Owegi, Margaret A, Quick, Adam, Wymer, James, Goutman, Stephen A, Heitzman, Daragh, Heiman-Patterson, Terry, Jackson, Carlayne E, Quinn, Colin, Rothstein, Jeffrey D, Kasarskis, Edward J, Katz, Jonathan, Jenkins, Liberty, Ladha, Shafeeq, Miller, Timothy M, Scelsa, Stephen N, Vu, Tuan H, Fournier, Christina N, Glass, Jonathan D, Johnson, Kristin M, Swenson, Andrea, Goyal, Namita A, Pattee, Gary L, Andres, Patricia L, Babu, Suma, Chase, Marianne, Dagostino, Derek, Dickson, Samuel P, Ellison, Noel, Hall, Meghan, Hendrix, Kent, Kittle, Gale, McGovern, Michelle, Ostrow, Joseph, Pothier, Lindsay, Randall, Rebecca, Shefner, Jeremy M, Sherman, Alexander V, Tustison, Eric, Vigneswaran, Prasha, Walker, Jason, Yu, Hong, Chan, James, Wittes, Janet, Cohen, Joshua, Klee, Justin, Leslie, Kent, Tanzi, Rudolph E, Gilbert, Walter, Yeramian, Patrick D, Schoenfeld, David, Cudkowicz, Merit E
Format:	Artikel
Sprache:	eng
Schlagworte:	Age Aged Amyotrophic lateral sclerosis Amyotrophic Lateral Sclerosis - drug therapy Death Design Disease Progression Double-Blind Method Double-blind studies Drug Combinations Drug dosages Female Humans Intention to Treat Analysis Male Middle Aged Muscle contraction Muscle strength Neurofilaments Ostomy Pharmaceuticals Phenylbutyrates - adverse effects Phenylbutyrates - therapeutic use Phenylbutyric acid Severity of Illness Index Sodium Taurochenodeoxycholic Acid - administration & dosage Taurochenodeoxycholic Acid - therapeutic use Tracheostomy Tracheotomy Treatment Outcome
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Beschreibung
Zusammenfassung:	Sodium phenylbutyrate combined with taurursodiol reduces neuronal endoplasmic reticulum stress and mitochondrial dysfunction in experimental models. In a randomized trial, the combination slowed the rate of progression of ALS but did not affect the slow vital capacity or isometric muscle strength.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1916945