Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design
Identifieur interne : 002558 ( Main/Merge ); précédent : 002557; suivant : 002559Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design
Auteurs : Lisa S. Friedman [États-Unis] ; Jennifer M. Farmer [États-Unis] ; Susan Perlman [États-Unis] ; George Wilmot [États-Unis] ; Christopher M. Gomez [États-Unis] ; Khalaf O. Bushara [États-Unis] ; Katherine D. Mathews [États-Unis] ; S. H. Subramony [États-Unis] ; Tetsuo Ashizawa [États-Unis] ; Laura J. Balcer [États-Unis] ; Robert B. Wilson [États-Unis] ; David R. Lynch [États-Unis]Source :
- Movement disorders [ 0885-3185 ] ; 2010.
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- Pascal (Inist)
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- topic : Neurologie.
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Abstract
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.
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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en" level="a">Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles</s1><s2>Los Angeles, California</s2><s3>USA</s3><sZ>3 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Department of Neurology, Emory University</s1><s2>Atlanta, Georgia</s2><s3>USA</s3><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Géorgie (États-Unis)</region></placeName></affiliation></author><author><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1><s2>Minneapolis, Minnesota</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Chicago</s1><s2>Chicago, Illinois</s2><s3>USA</s3><sZ>5 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Illinois</region></placeName></affiliation></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O." last="Bushara">Khalaf O. Bushara</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1><s2>Minneapolis, Minnesota</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><inist:fA14 i1="08"><s1>Departments of Neurology and Pediatrics, University of Iowa</s1><s2>Iowa City, Iowa</s2><s3>USA</s3><sZ>7 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1><s2>Galveston, Texas</s2><s3>USA</s3><sZ>8 aut.</sZ><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Mississippi</s1><s2>Jackson, Mississippi</s2><s3>USA</s3><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1><s2>Galveston, Texas</s2><s3>USA</s3><sZ>8 aut.</sZ><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Florida</s1><s2>Gainesville, Florida</s2><s3>USA</s3><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name><affiliation wicri:level="2"><inist:fA14 i1="12"><s1>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>11 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">INIST</idno><idno type="inist">10-0178204</idno><date when="2010">2010</date><idno type="stanalyst">PASCAL 10-0178204 INIST</idno><idno type="RBID">Pascal:10-0178204</idno><idno type="wicri:Area/PascalFrancis/Corpus">000C17</idno><idno type="wicri:Area/PascalFrancis/Curation">002102</idno><idno type="wicri:Area/PascalFrancis/Checkpoint">000971</idno><idno type="wicri:doubleKey">0885-3185:2010:Friedman L:measuring:the:rate</idno><idno type="wicri:Area/Main/Merge">002558</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design</title><author><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><affiliation wicri:level="2"><inist:fA14 i1="04"><s1>Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles</s1><s2>Los Angeles, California</s2><s3>USA</s3><sZ>3 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Californie</region></placeName></affiliation></author><author><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><affiliation wicri:level="2"><inist:fA14 i1="05"><s1>Department of Neurology, Emory University</s1><s2>Atlanta, Georgia</s2><s3>USA</s3><sZ>4 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Géorgie (États-Unis)</region></placeName></affiliation></author><author><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1><s2>Minneapolis, Minnesota</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="07"><s1>Department of Neurology, University of Chicago</s1><s2>Chicago, Illinois</s2><s3>USA</s3><sZ>5 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Illinois</region></placeName></affiliation></author><author><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O." last="Bushara">Khalaf O. Bushara</name><affiliation wicri:level="2"><inist:fA14 i1="06"><s1>Department of Neurology, University of Minnesota</s1><s2>Minneapolis, Minnesota</s2><s3>USA</s3><sZ>5 aut.</sZ><sZ>6 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Minnesota</region></placeName></affiliation></author><author><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name><affiliation wicri:level="4"><inist:fA14 i1="08"><s1>Departments of Neurology and Pediatrics, University of Iowa</s1><s2>Iowa City, Iowa</s2><s3>USA</s3><sZ>7 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Iowa</region><settlement type="city">Iowa City</settlement></placeName><orgName type="university">Université de l'Iowa</orgName></affiliation></author><author><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1><s2>Galveston, Texas</s2><s3>USA</s3><sZ>8 aut.</sZ><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Department of Neurology, University of Mississippi</s1><s2>Jackson, Mississippi</s2><s3>USA</s3><sZ>8 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">État du Mississippi</region></placeName></affiliation></author><author><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><affiliation wicri:level="2"><inist:fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1><s2>Galveston, Texas</s2><s3>USA</s3><sZ>8 aut.</sZ><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Texas</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="11"><s1>Department of Neurology, University of Florida</s1><s2>Gainesville, Florida</s2><s3>USA</s3><sZ>9 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Floride</region></placeName></affiliation></author><author><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name><affiliation wicri:level="2"><inist:fA14 i1="12"><s1>Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>11 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation></author><author><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><affiliation wicri:level="2"><inist:fA14 i1="01"><s1>Department of Neurology, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>10 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</region></placeName></affiliation><affiliation wicri:level="2"><inist:fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1><s2>Philadelphia, Pennsylvania</s2><s3>USA</s3><sZ>1 aut.</sZ><sZ>2 aut.</sZ><sZ>12 aut.</sZ></inist:fA14><country>États-Unis</country><placeName><region type="state">Pennsylvanie</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>Ataxia</term><term>Clinical trial</term><term>Friedreich ataxia</term><term>Mitochondrial disorder</term><term>Nervous system diseases</term><term>Neurology</term><term>Trinucleotide</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Hérédodégénérescence spinocérébelleuse de Friedreich</term><term>Ataxie</term><term>Cytopathie mitochondriale</term><term>Pathologie du système nerveux</term><term>Essai clinique</term><term>Neurologie</term><term>Trinucléotide</term></keywords><keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Neurologie</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia of all four limbs, dysarthria, and arreflexia. A variety of measures are currently used to quantify disease progression, including the Friedreich Ataxia Rating Scale, examiner-rated functional disability scales, self-reported activities of daily living and performance measures such as the timed 25-foot walk, 9-hole pegboard test, PATA speech test, and low-contrast letter acuity vision charts. This study examines the rate of disease progression over one and two years in a cohort of 236 Friedreich ataxia patients using these scales and performance measure composites. The Friedreich Ataxia Rating Scale and performance-measure composites captured disease progression, with a greater sensitivity to change over 2 years than over 1 year. The measures differed in their sensitivity to change and in possible bias. These results help to establish norms for progression in FRDA that can be useful in measuring the long-term success of therapeutic agents and defining sample-size calculations for double-blind clinical trials.</div></front></TEI><affiliations><list><country><li>États-Unis</li></country><region><li>Californie</li><li>Floride</li><li>Géorgie (États-Unis)</li><li>Illinois</li><li>Iowa</li><li>Minnesota</li><li>Pennsylvanie</li><li>Texas</li><li>État du Mississippi</li></region><settlement><li>Iowa City</li></settlement><orgName><li>Université de l'Iowa</li></orgName></list><tree><country name="États-Unis"><region name="Pennsylvanie"><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name></region><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><name sortKey="Ashizawa, Tetsuo" sort="Ashizawa, Tetsuo" uniqKey="Ashizawa T" first="Tetsuo" last="Ashizawa">Tetsuo Ashizawa</name><name sortKey="Balcer, Laura J" sort="Balcer, Laura J" uniqKey="Balcer L" first="Laura J." last="Balcer">Laura J. Balcer</name><name sortKey="Bushara, Khalaf O" sort="Bushara, Khalaf O" uniqKey="Bushara K" first="Khalaf O." last="Bushara">Khalaf O. Bushara</name><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><name sortKey="Farmer, Jennifer M" sort="Farmer, Jennifer M" uniqKey="Farmer J" first="Jennifer M." last="Farmer">Jennifer M. Farmer</name><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name><name sortKey="Friedman, Lisa S" sort="Friedman, Lisa S" uniqKey="Friedman L" first="Lisa S." last="Friedman">Lisa S. Friedman</name><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name><name sortKey="Gomez, Christopher M" sort="Gomez, Christopher M" uniqKey="Gomez C" first="Christopher M." last="Gomez">Christopher M. Gomez</name><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><name sortKey="Lynch, David R" sort="Lynch, David R" uniqKey="Lynch D" first="David R." last="Lynch">David R. Lynch</name><name sortKey="Mathews, Katherine D" sort="Mathews, Katherine D" uniqKey="Mathews K" first="Katherine D." last="Mathews">Katherine D. Mathews</name><name sortKey="Perlman, Susan" sort="Perlman, Susan" uniqKey="Perlman S" first="Susan" last="Perlman">Susan Perlman</name><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><name sortKey="Subramony, S H" sort="Subramony, S H" uniqKey="Subramony S" first="S. H." last="Subramony">S. H. Subramony</name><name sortKey="Wilmot, George" sort="Wilmot, George" uniqKey="Wilmot G" first="George" last="Wilmot">George Wilmot</name><name sortKey="Wilson, Robert B" sort="Wilson, Robert B" uniqKey="Wilson R" first="Robert B." last="Wilson">Robert B. Wilson</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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