Voice Onset Time in Early- and Late-Stage Amyotrophic Lateral Sclerosis

Autor: Anusha Thomas, Kristin J. Teplansky, Alan Wisler, Daragh Heitzman, Sara Austin, Jun Wang

Publikováno v: J Speech Lang Hear Res

Purpose: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects bulbar functions including speech and voice. Voice onset time (VOT) was examined in speakers with ALS in early and late stages to explore the coordination of the

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::380089059adc49b9c34b65f330898c25
https://doi.org/10.1044/2022_jslhr-21-00632

Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

Autor: Emily G. Baxi, Terri Thompson, Jonathan Li, Julia A. Kaye, Ryan G. Lim, Jie Wu, Divya Ramamoorthy, Leandro Lima, Vineet Vaibhav, Andrea Matlock, Aaron Frank, Alyssa N. Coyne, Barry Landin, Loren Ornelas, Elizabeth Mosmiller, Sara Thrower, S. Michelle Farr, Lindsey Panther, Emilda Gomez, Erick Galvez, Daniel Perez, Imara Meepe, Susan Lei, Berhan Mandefro, Hannah Trost, Louis Pinedo, Maria G. Banuelos, Chunyan Liu, Ruby Moran, Veronica Garcia, Michael Workman, Richie Ho, Stacia Wyman, Jennifer Roggenbuck, Matthew B. Harms, Jennifer Stocksdale, Ricardo Miramontes, Keona Wang, Vidya Venkatraman, Ronald Holewenski, Niveda Sundararaman, Rakhi Pandey, Danica-Mae Manalo, Aneesh Donde, Nhan Huynh, Miriam Adam, Brook T. Wassie, Edward Vertudes, Naufa Amirani, Krishna Raja, Reuben Thomas, Lindsey Hayes, Alex Lenail, Aianna Cerezo, Sarah Luppino, Alanna Farrar, Lindsay Pothier, Carolyn Prina, Todd Morgan, Arish Jamil, Sarah Heintzman, Jennifer Jockel-Balsarotti, Elizabeth Karanja, Jesse Markway, Molly McCallum, Ben Joslin, Deniz Alibazoglu, Stephen Kolb, Senda Ajroud-Driss, Robert Baloh, Daragh Heitzman, Tim Miller, Jonathan D. Glass, Natasha Leanna Patel-Murray, Hong Yu, Ervin Sinani, Prasha Vigneswaran, Alexander V. Sherman, Omar Ahmad, Promit Roy, Jay C. Beavers, Steven Zeiler, John W. Krakauer, Carla Agurto, Guillermo Cecchi, Mary Bellard, Yogindra Raghav, Karen Sachs, Tobias Ehrenberger, Elizabeth Bruce, Merit E. Cudkowicz, Nicholas Maragakis, Raquel Norel, Jennifer E. Van Eyk, Steven Finkbeiner, James Berry, Dhruv Sareen, Leslie M. Thompson, Ernest Fraenkel, Clive N. Svendsen, Jeffrey D. Rothstein

Publikováno v: Nature Neuroscience. 25:226-237

Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent stem (iPS) cell lines, multi-omic data derived from iPS neurons and longitudinal clinical and smartphone data from over 1,000 patients with ALS. This resource pr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7bcf0bf833d3caf459c35b182e837fc5
https://doi.org/10.1038/s41593-021-01006-0

Safety and efficacy of oral levosimendan in people with amyotrophic lateral sclerosis (the REFALS study): a randomised, double-blind, placebo-controlled phase 3 trial

Autor: Michael Pulley, Mikko Kuoppamäki, Carolyn A Young, Jesus S. Mora Pardina, Kumaraswamy Sivakumar, Toni Sarapohja, Michael A. Elliott, Chafic Karam, Sandeep Rana, Orla Hardiman, Nathan P. Staff, Letizia Mazzini, Gabriele Mora, Thomas F. Meyer, Colleen O'Connell, Stéphanie Delstanche, Elham Bayat, Michael D. Weiss, Waqar Waheed, Nenad Mitrovic, Philippe Corcia, Marie-Hélène Soriani, Edward J. Kasarskis, Claudia Caponnetto, Dale J. Lange, Tuan Vu, Leo McCluskey, Berthold Schrank, Angela Genge, Matthew C. Kiernan, Valtteri V Aho, Manu Jokela, Philip Van Damme, Juan F. Vázquez Costa, Maurizio Inghilleri, Wolfgang Löscher, David Schultz, Tero Tapiola, Susanne Petri, Adriano Chiò, Gary L. Pattee, Julian Großkreutz, Ammar Al-Chalabi, Aziz Shaibani, Susan Mathers, Kevin J. Felice, Kimberly Goslin, James Caress, Matthias Boentert, Albert C. Ludolph, Aleksandar Radunovic, Robert D. Henderson, James Wymer, Todd Levine, Jakob Rath, Merrilee Needham, William Camu, Gaurav Guliani, Rune Johansson, Leonard H. van den Berg, Namita Goyal, Mark B. Bromberg, Bjorn Oskarsson, Annie Dionne, Eduardo Locatelli, Brent T. Harris, Suma Babu, Richard Bedlack, John Ravits, Jinsy A. Andrews, Philippe Couratier, Gabriele Siciliano, Hannu Laaksovirta, Kourosh Rezania, Lawrence Korngut, Eduardo Aguera Morales, Peter M Andersen, Eva Farrero Munoz, David Lacomis, Stephen N. Scelsa, Chris Garratt, Matthew Burford, Merit Cudkowicz, Nicholas J. Maragakis, Wendy Johnston, Martin M. Brown, Johannes Prudlo, Justin Y. Kwan, Dominic B. Fee, Senda Ajroud-Driss, Stephen A. Goutman, John Turnbull, Michael H. Rivner, Timothy M. Miller, Jan De Bleecker, Caroline Ingre, Luis Varona, Genevieve Matte, Daragh Heitzman, Robert Untucht, Lorne Zinman, Adam Quick, Jonathan S. Katz

Publikováno v: The Lancet Neurology. 20:821-831

Summary Background There is an urgent unmet need for new therapies in amyotrophic lateral sclerosis. In a clinical study with healthy volunteers, levosimendan, a calcium sensitiser, was shown to improve neuromechanical efficiency and contractile func

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::47e159b8bed1a737713c1c2f5bf0a571
https://doi.org/10.1016/s1474-4422(21)00242-8

Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis

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

Publikováno v: Muscle & Nerve

An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long�

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c577e9ab26d1e9b80fcf0903d01570f
https://doi.org/10.1002/mus.27091

Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial

Autor: Sabrina Paganoni, Suzanne Hendrix, Samuel P Dickson, Newman Knowlton, James D Berry, Michael A Elliott, Samuel Maiser, Chafic Karam, James B Caress, Margaret Ayo Owegi, Adam Quick, James Wymer, Stephen A Goutman, Daragh Heitzman, Terry D Heiman-Patterson, Carlayne Jackson, Colin Quinn, Jeffrey D Rothstein, Edward J Kasarskis, Jonathan Katz, Liberty Jenkins, Shafeeq S Ladha, Timothy M Miller, Stephen N Scelsa, Tuan H Vu, Christina Fournier, Kristin M Johnson, Andrea Swenson, Namita Goyal, Gary L Pattee, Suma Babu, Marianne Chase, Derek Dagostino, Meghan Hall, Gale Kittle, Mathew Eydinov, Joseph Ostrow, Lindsay Pothier, Rebecca Randall, Jeremy M Shefner, Alexander V Sherman, Eric Tustison, Prasha Vigneswaran, Hong Yu, Joshua Cohen, Justin Klee, Rudolph Tanzi, Walter Gilbert, Patrick Yeramian, Merit Cudkowicz

Publikováno v: Journal of neurology, neurosurgery, and psychiatry, vol 93, iss 8

BackgroundCoformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) was shown to prolong survival and slow functional decline in amyotrophic lateral sclerosis (ALS).ObjectiveDetermine whether PB/TURSO prolonged tracheostomy/ventilation-free survival

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::668333e30d83e12f19bd834ace7127dc
https://pubmed.ncbi.nlm.nih.gov/35577511