Present and future of disease-modifying therapies in multiple system atrophy.
Autonomic Neuroscience. 2018-05-01; 211: 31-38
DOI: 10.1016/j.autneu.2017.12.008
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1. Auton Neurosci. 2018 May;211:31-38. doi: 10.1016/j.autneu.2017.12.008. Epub 2018
Jan 4.
Present and future of disease-modifying therapies in multiple system atrophy.
Lopez-Cuina M(1), Foubert-Samier A(2), Tison F(2), Meissner WG(3).
Author information:
(1)Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000
Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293,
F-33000 Bordeaux, France.
(2)Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000
Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293,
F-33000 Bordeaux, France; Centre de Référence Maladie Rare AMS, Service de
Neurologie, Hôpital Pellegrin, CHU de Bordeaux, F-33000 Bordeaux, France.
(3)Univ. de Bordeaux, Institut des Maladies Neurodégénératives, UMR 5293, F-33000
Bordeaux, France; CNRS, Institut des Maladies Neurodégénératives, UMR 5293,
F-33000 Bordeaux, France; Centre de Référence Maladie Rare AMS, Service de
Neurologie, Hôpital Pellegrin, CHU de Bordeaux, F-33000 Bordeaux, France.
Electronic address: .
Through the last decade seven clinical trials on Multiple System Atrophy have
been published, virtually all of them reported negative results. Patients and
family remain hopeful while facing this devastating disease, but as doctors we
still cannot offer them disease-modifying therapies. The field has seen many
advances regarding pathophysiology, translational research, diagnostic accuracy,
natural history and imaging, but successful treatment remains elusive. This
review provides an overview of the available tools for designing clinical trials,
critically analyzes the past studies and describes the knowledge obtained from
them, and finally gives some orientation for future trials that could meet the
current needs of patients and clinicians, overcoming the hurdles met by previous
studies.
Copyright © 2018 Elsevier B.V. All rights reserved.
DOI: 10.1016/j.autneu.2017.12.008
PMID: 29311010