Defining Disease-Modifying Therapies for PD- A Road Map for Moving Forward
Identifieur interne : 001F59 ( Main/Exploration ); précédent : 001F58; suivant : 001F60Defining Disease-Modifying Therapies for PD- A Road Map for Moving Forward
Auteurs : C. Warren Olanow [États-Unis, Italie] ; Karl Kieburtz [États-Unis]Source :
- Movement disorders [ 0885-3185 ] ; 2010.
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- Pascal (Inist)
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Abstract
A disease-modifying therapy that slows or stops disease progression is one of the major unmet needs in the management of Parkinson's disease. To date, no therapy has been approved for disease modification despite promising laboratory data and positive results in clinical trials. This is because confounding symptomatic or pharmacologic effects cannot be excluded. The delayed start study provides an opportunity to define therapies that provide benefit that cannot be explained by an early symptomatic effect alone. However, this trial design does not necessarily provide a meaningful measure of the effect of the intervention on cumulative disability. In contrast, the long-term simple study provides a measure of the effect of the drug on cumulative disability but does not address mechanism of action. Together these two trials provide a road map for defining a disease modifying drug and determining the long term cumulative effect of the drug on the disease.
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Warren Olanow</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology and Neuroscience, Mount Sinai School of Medicine</s1><s2>New York, New York</s2><s3>USA</s3><sZ>1 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation><affiliation wicri:level="3"><inist:fA14 i1="02"><s1>Department of Neurology, IRCCS San Raffaele Pisana</s1><s2>Rome</s2><s3>ITA</s3><sZ>1 aut.</sZ></inist:fA14><country>Italie</country><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Kieburtz, Karl" sort="Kieburtz, Karl" uniqKey="Kieburtz K" first="Karl" last="Kieburtz">Karl Kieburtz</name><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Department of Neurology, University of Rochester</s1><s2>Rochester, New York</s2><s3>USA</s3><sZ>2 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">INIST</idno><idno type="inist">10-0446299</idno><date when="2010">2010</date><idno type="stanalyst">PASCAL 10-0446299 INIST</idno><idno type="RBID">Pascal:10-0446299</idno><idno type="wicri:Area/PascalFrancis/Corpus">000930</idno><idno type="wicri:Area/PascalFrancis/Curation">002389</idno><idno type="wicri:Area/PascalFrancis/Checkpoint">000B23</idno><idno type="wicri:doubleKey">0885-3185:2010:Olanow C:defining:disease:modifying</idno><idno type="wicri:Area/Main/Merge">002710</idno><idno type="wicri:Area/Main/Curation">001F59</idno><idno type="wicri:Area/Main/Exploration">001F59</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Defining Disease-Modifying Therapies for PD- A Road Map for Moving Forward</title><author><name sortKey="Olanow, C Warren" sort="Olanow, C Warren" uniqKey="Olanow C" first="C. Warren" last="Olanow">C. Warren Olanow</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology and Neuroscience, Mount Sinai School of Medicine</s1><s2>New York, New York</s2><s3>USA</s3><sZ>1 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation><affiliation wicri:level="3"><inist:fA14 i1="02"><s1>Department of Neurology, IRCCS San Raffaele Pisana</s1><s2>Rome</s2><s3>ITA</s3><sZ>1 aut.</sZ></inist:fA14><country>Italie</country><placeName><settlement type="city">Rome</settlement><region nuts="2">Latium</region></placeName></affiliation></author><author><name sortKey="Kieburtz, Karl" sort="Kieburtz, Karl" uniqKey="Kieburtz K" first="Karl" last="Kieburtz">Karl Kieburtz</name><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Department of Neurology, University of Rochester</s1><s2>Rochester, New York</s2><s3>USA</s3><sZ>2 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État de New York</region></placeName></affiliation></author></analytic><series><title level="j" type="main">Movement disorders</title><title level="j" type="abbreviated">Mov. disord.</title><idno type="ISSN">0885-3185</idno><imprint><date when="2010">2010</date></imprint></series></biblStruct></sourceDesc><seriesStmt><title level="j" type="main">Movement disorders</title><title level="j" type="abbreviated">Mov. disord.</title><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Cartography</term><term>Clinical trial</term><term>Long term</term><term>Modification</term><term>Nervous system diseases</term><term>Parkinson disease</term><term>Treatment</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Maladie de Parkinson</term><term>Pathologie du système nerveux</term><term>Traitement</term><term>Cartographie</term><term>Modification</term><term>Essai clinique</term><term>Long terme</term></keywords><keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Cartographie</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">A disease-modifying therapy that slows or stops disease progression is one of the major unmet needs in the management of Parkinson's disease. To date, no therapy has been approved for disease modification despite promising laboratory data and positive results in clinical trials. This is because confounding symptomatic or pharmacologic effects cannot be excluded. The delayed start study provides an opportunity to define therapies that provide benefit that cannot be explained by an early symptomatic effect alone. However, this trial design does not necessarily provide a meaningful measure of the effect of the intervention on cumulative disability. In contrast, the long-term simple study provides a measure of the effect of the drug on cumulative disability but does not address mechanism of action. Together these two trials provide a road map for defining a disease modifying drug and determining the long term cumulative effect of the drug on the disease.</div></front></TEI><affiliations><list><country><li>Italie</li><li>États-Unis</li></country><region><li>Latium</li><li>État de New York</li></region><settlement><li>Rome</li></settlement></list><tree><country name="États-Unis"><region name="État de New York"><name sortKey="Olanow, C Warren" sort="Olanow, C Warren" uniqKey="Olanow C" first="C. Warren" last="Olanow">C. Warren Olanow</name></region><name sortKey="Kieburtz, Karl" sort="Kieburtz, Karl" uniqKey="Kieburtz K" first="Karl" last="Kieburtz">Karl Kieburtz</name></country><country name="Italie"><region name="Latium"><name sortKey="Olanow, C Warren" sort="Olanow, C Warren" uniqKey="Olanow C" first="C. Warren" last="Olanow">C. Warren Olanow</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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