Randomized double-blind personalized N-of-1 clinical trial to test the safety and potential efficacy of TJ-68 for treating muscle cramps in amyotrophic lateral sclerosis (ALS): study protocol for a TJ-68 trial

Autor: Hiroshi Mitsumoto, Ken Cheung, Björn Oskarsson, Howard F. Andrews, Grace E. Jang, Jinsy A. Andrews, Jaimin S. Shah, Joseph Americo Fernandes, Martin McElhiney, Regina M. Santella

Publikováno v: Trials, Vol 24, Iss 1, Pp 1-17 (2023)

Abstract Introduction/aims Muscle cramps are a common and often disabling symptom in amyotrophic lateral sclerosis (ALS), a devastating and incurable neurodegenerative disorder. To date, there are no medications specifically approved for the treatmen

Externí odkaz: https://doaj.org/article/94c2ebdf0aeb44f6bf42c2508a51d2ee

Health utilities and quality-adjusted life years for patients with amyotrophic lateral sclerosis receiving reldesemtiv or placebo in FORTITUDE-ALS

Autor: Paulos Gebrehiwet, Lisa Meng, Stacy A. Rudnicki, Phil Sarocco, Jenny Wei, Andrew A. Wolff, Michael Butzner, Adriano Chiò, Jinsy A. Andrews, Angela Genge, Dyfrig A. Hughes, Carlayne E. Jackson, Noah Lechtzin, Timothy M. Miller, Jeremy M. Shefner

Publikováno v: Journal of Medical Economics. 26:488-493

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::24e54d5461c926b57c6ea7ac536ac146
https://doi.org/10.1080/13696998.2023.2192588

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

Medical therapies for amyotrophic lateral sclerosis-related respiratory decline: an appraisal of needs, opportunities and obstacles

Autor: Susana Pinto, Lora Clawson, Julian Grosskreutz, Orla Hardiman, Carolyn A Young, Jinsy A. Andrews, Merit Cudkowicz

Publikováno v: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 23:66-75

A roundtable convened in July 2020 examined issues concerning respiratory support in amyotrophic lateral sclerosis (ALS), with reference to the potential for an early-phase orally administered medication that might either postpone the introduction of

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::02d077657ad82b351acdee993c037d31
https://doi.org/10.1080/21678421.2021.1920981

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

Autor: Gary L. Pattee, Ashley Whyte-Rayson, Andrew A. Wolff, Jeremy M. Shefner, Lisa Meng, Jesus S. Mora, Lorne Zinman, Steve Vucic, Terry Heiman-Patterson, Stephen J. Kolb, James Caress, Bettina M. Cockroft, Carlayne E. Jackson, Timothy M. Miller, Michael D. Weiss, Ghazala Hayat, Shumaila Sultan, Benjamin Rix Brooks, Daragh Heitzman, Tuan Vu, Merrilee Needham, Dianna Quan, Genevieve Matte, Shafeeq Ladha, Orla Hardiman, Fady I. Malik, Zachary Simmons, Wendy Johnston, Christen Shoesmith, Namita Goyal, Erik P. Pioro, James Wymer, David Schultz, Leonard H. van den Berg, Cynthia Bodkin, Lawrence Korngut, Jeffrey Statland, Michael Pulley, Bjorn Oskarsson, Chafic Karam, Angela Genge, Matthew C. Kiernan, Jenny Wei, Annie Dionne, Jinsy A. Andrews, Noah Lechtzin, Stephen A. Goutman, Andrea Swenson, Dominic B. Fee, Kerri Schellenberg, Robert D. Henderson, Kourosh Rezania, Stacy A. Rudnicki

Publikováno v: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. 22:287-299

To evaluate safety, dose response, and preliminary efficacy of reldesemtiv over 12 weeks in patients with amyotrophic lateral sclerosis (ALS). Methods: Patients (≤2 years since diagnosis) with slow...

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::4962fd94da927caf368e57b8248abdd4
https://doi.org/10.1080/21678421.2020.1822410