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

Novel Mutation in CACNA1A Associated with Activity-Induced Dystonia, Cervical Dystonia, and Mild Ataxia

Autor: Benjamin Stampfl, Dominic B. Fee

Publikováno v: Case Reports in Neurological Medicine
Case Reports in Neurological Medicine, Vol 2021 (2021)

CACNA1A encodes the pore-forming α1 subunit of the neuronal voltage-gated Cav2.1 (P/Q-type) channels, which are predominantly localized at the presynaptic terminals of the brain and cerebellar neurons and play an important role in controlling neurot

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9dfdf0ec766de6153065eaf594b1c60b
http://europepmc.org/articles/PMC8356012

Skill, confidence and support: conceptual elements of a child/youth caregiver training program in amyotrophic lateral sclerosis - the YCare protocol

Autor: Melinda S. Kavanaugh, Dominic B. Fee, Young Ik Cho, Paul E. Barkhaus

Publikováno v: Neurodegenerative disease management. 10(4)

Aim: Pilot study of a novel caregiving training and support intervention for children and youth

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ef93fbfbc64212777a762f53e3dfcf8
https://pubmed.ncbi.nlm.nih.gov/32749175

Siblings With Mutations in TRAPPC11 Presenting With Limb-Girdle Muscular Dystrophy 2S

Autor: Priya Monrad, Matthew Harmelink, Dominic B. Fee, Erika Pyzik

Publikováno v: Journal of Clinical Neuromuscular Disease. 19:27-30

Limb-girdle muscular dystrophy 2S (LGMD2S) is an autosomal recessive condition due to mutations in the TRAPPC11 gene. It is recently described with only 9 prior reported individuals. In addition to the muscular dystrophy, some affected individuals ha

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6960eee4d3d08711d4de04078815e374
https://doi.org/10.1097/cnd.0000000000000173

Performance evaluation of MOMA (MOment MAtching) – a remote network calibration technique for PM2.5 and PM10 sensors

Autor: L. F. Weissert, G. S. Henshaw, D. E. Williams, B. Feenstra, R. Lam, A. Collier-Oxandale, V. Papapostolou, A. Polidori

Publikováno v: Atmospheric Measurement Techniques, Vol 16, Pp 4709-4722 (2023)

We evaluate the potential of using a previously developed remote calibration framework we name MOMA (MOment MAtching) to improve the data quality in particulate matter (PM) sensors deployed in hierarchical networks. MOMA assumes that a network of ref

Externí odkaz: https://doaj.org/article/2db14b1923ea46d6a57cc298c902dd0e