Literature DB >> 26515630

The Dilemma of the Clinical Trialist in Amyotrophic Lateral Sclerosis: The Hurdles to Finding a Cure.

Jonathan S Katz1, Richard J Barohn2, Mazen M Dimachkie2, Hiroshi Mitsumoto3.   

Abstract

Amyotrophic lateral sclerosis can be described as a disease with a poorly understood pathophysiologic mechanism and no treatment that dramatically impacts the course of the disease. Clinical trialists are faced with finding small treatment effects against a background of multiple potential treatments, a past history of failed trials, and heterogenous clinical outcomes. This article summarizes this environment and provides a rationale for drug development going forward.
Copyright © 2015 Elsevier Inc. All rights reserved.

Entities:  

Keywords:  Amyotrophic lateral sclerosis; Clinical trialists; Drug development; Treatment

Mesh：

Year:  2015        PMID： 26515630     DOI： 10.1016/j.ncl.2015.07.014

Source DB:  PubMed          Journal:  Neurol Clin        ISSN： 0733-8619            Impact factor:   3.806


  4 in total

1.  Integrative Profiling of Amyotrophic Lateral Sclerosis Lymphoblasts Identifies Unique Metabolic and Mitochondrial Disease Fingerprints.

Authors:  Teresa Cunha-Oliveira; Marcelo Carvalho; Vilma Sardão; Elisabete Ferreiro; Débora Mena; Francisco B Pereira; Fernanda Borges; Paulo J Oliveira; Filomena S G Silva
Journal:  Mol Neurobiol       Date:  2022-08-06       Impact factor: 5.682

2.  Delayed onset of inherited ALS by deletion of the BDNF receptor TrkB.T1 is non-cell autonomous.

Authors:  Sudhirkumar Yanpallewar; Gianluca Fulgenzi; Francesco Tomassoni-Ardori; Colleen Barrick; Lino Tessarollo
Journal:  Exp Neurol       Date:  2020-12-24       Impact factor: 5.620

3.  A Phase 2A randomized, double-blind, placebo-controlled pilot trial of GM604 in patients with Amyotrophic Lateral Sclerosis (ALS Protocol GALS-001) and a single compassionate patient treatment (Protocol GALS-C).

Authors:  Mark Kindy; Paul Lupinacci; Raymond Chau; Tony Shum; Dorothy Ko
Journal:  F1000Res       Date:  2017-03-07

4.  Fibroblast bioenergetics to classify amyotrophic lateral sclerosis patients.

Authors:  Csaba Konrad; Hibiki Kawamata; Kirsten G Bredvik; Andrea J Arreguin; Steven A Cajamarca; Jonathan C Hupf; John M Ravits; Timothy M Miller; Nicholas J Maragakis; Chadwick M Hales; Jonathan D Glass; Steven Gross; Hiroshi Mitsumoto; Giovanni Manfredi
Journal:  Mol Neurodegener       Date:  2017-10-24       Impact factor: 14.195
  4 in total

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