Drugs and clinical trials in neurodegenerative diseases

Neurodegenerative diseases are disabling conditions continuously increasing due to aging of population. A disease modifying therapy that slows or stops disease progression is therefore a major unmet medical need. Unfortunately, research for effective treatments is hampered by lack of knowledge on th...

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Main Authors: Paolo Stanzione, Domenicantonio Tropepi
Format: Article
Language:English
Published: Istituto Superiore di Sanità 2011-01-01
Series:Annali dell'Istituto Superiore di Sanità
Subjects:
Online Access:http://www.scielosp.org/scielo.php?script=sci_arttext&pid=S0021-25712011000100011&lng=en&tlng=en
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author Paolo Stanzione
Domenicantonio Tropepi
author_facet Paolo Stanzione
Domenicantonio Tropepi
author_sort Paolo Stanzione
collection DOAJ
description Neurodegenerative diseases are disabling conditions continuously increasing due to aging of population. A disease modifying therapy that slows or stops disease progression is therefore a major unmet medical need. Unfortunately, research for effective treatments is hampered by lack of knowledge on the pathologic processes underpinning these diseases and of reliable biomarkers. Clinical trials are difficult, as they require large populations that need to be followed for very long periods to capture possible effects on disease progression. These difficulties produce frequent failures and waste of human and economic resources. Since research has to continue in this area, until comprehensive knowledge of basic pathologic processes is obtained, alternative study designs can be considered to identify disease modifiers and to reduce costs of clinical studies.
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spelling doaj.art-22812d8f640546f2815666720e08b58b2022-12-21T17:14:23ZengIstituto Superiore di SanitàAnnali dell'Istituto Superiore di Sanità0021-25712011-01-01471495410.4415/ANN_11_01_11S0021-25712011000100011Drugs and clinical trials in neurodegenerative diseasesPaolo Stanzione0Domenicantonio Tropepi1Università degli Studi Tor VergataIRCCS Fondazione S. LuciaNeurodegenerative diseases are disabling conditions continuously increasing due to aging of population. A disease modifying therapy that slows or stops disease progression is therefore a major unmet medical need. Unfortunately, research for effective treatments is hampered by lack of knowledge on the pathologic processes underpinning these diseases and of reliable biomarkers. Clinical trials are difficult, as they require large populations that need to be followed for very long periods to capture possible effects on disease progression. These difficulties produce frequent failures and waste of human and economic resources. Since research has to continue in this area, until comprehensive knowledge of basic pathologic processes is obtained, alternative study designs can be considered to identify disease modifiers and to reduce costs of clinical studies.http://www.scielosp.org/scielo.php?script=sci_arttext&pid=S0021-25712011000100011&lng=en&tlng=enmalattie neurodegenerativedisease modifierdelayed start designrandomized withdrawal designstudio di futilità
spellingShingle Paolo Stanzione
Domenicantonio Tropepi
Drugs and clinical trials in neurodegenerative diseases
Annali dell'Istituto Superiore di Sanità
malattie neurodegenerative
disease modifier
delayed start design
randomized withdrawal design
studio di futilità
title Drugs and clinical trials in neurodegenerative diseases
title_full Drugs and clinical trials in neurodegenerative diseases
title_fullStr Drugs and clinical trials in neurodegenerative diseases
title_full_unstemmed Drugs and clinical trials in neurodegenerative diseases
title_short Drugs and clinical trials in neurodegenerative diseases
title_sort drugs and clinical trials in neurodegenerative diseases
topic malattie neurodegenerative
disease modifier
delayed start design
randomized withdrawal design
studio di futilità
url http://www.scielosp.org/scielo.php?script=sci_arttext&pid=S0021-25712011000100011&lng=en&tlng=en
work_keys_str_mv AT paolostanzione drugsandclinicaltrialsinneurodegenerativediseases
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