ParkinsonFranceV1, Hal, Corpus, bibRecord, 000768

Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.

Identifieur interne : 000768 ( Hal/Corpus ); précédent : 000767; suivant : 000769

Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.

Auteurs : Pierre Olivier Fernagut ; Benjamin Dehay ; Aline Maillard ; Erwan Bezard ; Paul Perez ; Anne Pavy-Le Traon ; Olivier Rascol ; Alexandra Foubert-Samier ; François Tison ; Wassilios Meissner

Source :

Neurobiology of Disease [ 0969-9961 ] ; 2014-07.

RBID : Hal:hal-01217333

Abstract

Despite active fundamental, translational and clinical research, no therapeutic intervention has yet shown convincing effects on disease progression in Parkinson's disease (PD) patients. Indeed, several disease-modification trials failed or proved to be inconclusive due to lack of consistency between clinical rating scales and putative surrogate markers of disease progression, or confounding symptomatic effects of the tested compound. Multiple system atrophy (MSA) is a rapidly progressing orphan disorder leading to severe motor disability within a few years. Together with PD and dementia with Lewy bodies (DLB), MSA belongs to the synucleinopathies, a group of neurodegenerative disorders characterized by the abnormal accumulation of alpha-synuclein. Crucial milestones have been reached for successfully conducting clinical intervention trials in a large number of patients with MSA. In this personal view, we will review evidence, and discuss why MSA could prove the most relevant clinical model for assessing treatments that target mechanisms operating in all synucleinopathies.

Url:

https://hal.archives-ouvertes.fr/hal-01217333

DOI: 10.1016

Links to Exploration step

Hal:hal-01217333

Le document en format XML

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<front><div type="abstract" xml:lang="en">Despite active fundamental, translational and clinical research, no therapeutic intervention has yet shown convincing effects on disease progression in Parkinson's disease (PD) patients. Indeed, several disease-modification trials failed or proved to be inconclusive due to lack of consistency between clinical rating scales and putative surrogate markers of disease progression, or confounding symptomatic effects of the tested compound. Multiple system atrophy (MSA) is a rapidly progressing orphan disorder leading to severe motor disability within a few years. Together with PD and dementia with Lewy bodies (DLB), MSA belongs to the synucleinopathies, a group of neurodegenerative disorders characterized by the abnormal accumulation of alpha-synuclein. Crucial milestones have been reached for successfully conducting clinical intervention trials in a large number of patients with MSA. In this personal view, we will review evidence, and discuss why MSA could prove the most relevant clinical model for assessing treatments that target mechanisms operating in all synucleinopathies.</div>
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            <abstract xml:lang="en">Despite active fundamental, translational and clinical research, no therapeutic intervention has yet shown convincing effects on disease progression in Parkinson's disease (PD) patients. Indeed, several disease-modification trials failed or proved to be inconclusive due to lack of consistency between clinical rating scales and putative surrogate markers of disease progression, or confounding symptomatic effects of the tested compound. Multiple system atrophy (MSA) is a rapidly progressing orphan disorder leading to severe motor disability within a few years. Together with PD and dementia with Lewy bodies (DLB), MSA belongs to the synucleinopathies, a group of neurodegenerative disorders characterized by the abnormal accumulation of alpha-synuclein. Crucial milestones have been reached for successfully conducting clinical intervention trials in a large number of patients with MSA. In this personal view, we will review evidence, and discuss why MSA could prove the most relevant clinical model for assessing treatments that target mechanisms operating in all synucleinopathies.</abstract>
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   |texte=   Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.
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	La maladie de Parkinson en France (serveur d'exploration)
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La maladie de Parkinson en France (serveur d'exploration)

Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.

Multiple system atrophy: a prototypical synucleinopathy for disease-modifying therapeutic strategies.

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