Improving clinical trial outcomes in amyotrophic lateral sclerosis

MC Kiernan, S Vucic, K Talbot, CJ McDermott… - Nature Reviews …, 2021 - nature.com
Individuals who are diagnosed with amyotrophic lateral sclerosis (ALS) today face the same
historically intransigent problem that has existed since the initial description of the disease in …

Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

H Mitsumoto, BR Brooks, V Silani - The Lancet Neurology, 2014 - thelancet.com
Amyotrophic lateral sclerosis (ALS) is one of the most rapidly progressive
neurodegenerative diseases of unknown cause. Riluzole is the only drug that slows disease …

Motor unit number estimation: a technology and literature review

CL Gooch, TJ Doherty, KM Chan, MB Bromberg… - Muscle & …, 2014 - Wiley Online Library
Introduction: Numerous methods for motor unit number estimation (MUNE) have been
developed. The objective of this article is to summarize and compare the major methods and …

Tracking a fast-moving disease: longitudinal markers, monitoring, and clinical trial endpoints in ALS

RH Chipika, E Finegan, S Li Hi Shing… - Frontiers in …, 2019 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) encompasses a heterogeneous group of phenotypes
with different progression rates, varying degree of extra-motor involvement and divergent …

Survival prediction in Amyotrophic lateral sclerosis based on MRI measures and clinical characteristics

C Schuster, O Hardiman, P Bede - BMC neurology, 2017 - Springer
Background Amyotrophic lateral sclerosis (ALS) a highly heterogeneous neurodegenerative
condition. Accurate diagnostic, monitoring and prognostic biomarkers are urgently needed …

Innovating clinical trials for amyotrophic lateral sclerosis: challenging the established order

RPA van Eijk, S Nikolakopoulos, KCB Roes, L Kendall… - Neurology, 2021 - AAN Enterprises
Development of effective treatments for amyotrophic lateral sclerosis (ALS) has been
hampered by disease heterogeneity, a limited understanding of underlying pathophysiology …

A randomized, placebo-controlled trial of memantine for functional disability in amyotrophic lateral sclerosis

M De Carvalho, S Pinto, J Costa… - Amyotrophic Lateral …, 2010 - Taylor & Francis
Our objective is to describe the results of a phase II/III, 12-months, double-blinded, single-
centre, randomized, parallel (1: 1), clinical trial performed to evaluate the efficacy and safety …

Age at onset influences on wide-ranged clinical features of sporadic amyotrophic lateral sclerosis

N Atsuta, H Watanabe, M Ito, F Tanaka… - Journal of the …, 2009 - Elsevier
PURPOSE: To profile the detailed clinical features of sporadic amyotrophic lateral sclerosis
(ALS) on large-scale samples in Japan. METHODS: We assessed the clinical features of …

Prognostic factors for the course of functional status of patients with ALS: a systematic review

H Creemers, H Grupstra, F Nollet, LH van den Berg… - Journal of …, 2015 - Springer
The progressive course of amyotrophic lateral sclerosis (ALS) results in an ever-changing
spectrum of the care needs of patients with ALS. Knowledge of prognostic factors for the …

Advancing drug discovery using the power of the human genome

K Heilbron, SV Mozaffari, V Vacic, P Yue… - The Journal of …, 2021 - Wiley Online Library
Human genetics plays an increasingly important role in drug development and population
health. Here we review the history of human genetics in the context of accelerating the …