MovDisordV3, PascalFrancis, Corpus, bibRecord, 000C17

Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design

Identifieur interne : 000C17 ( PascalFrancis/Corpus ); précédent : 000C16; suivant : 000C18

Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design

Auteurs : Lisa S. Friedman ; Jennifer M. Farmer ; Susan Perlman ; George Wilmot ; Christopher M. Gomez ; Khalaf O. Bushara ; Katherine D. Mathews ; S. H. Subramony ; Tetsuo Ashizawa ; Laura J. Balcer ; Robert B. Wilson ; David R. Lynch

Source :

Movement disorders [ 0885-3185 ] ; 2010.

RBID : Pascal:10-0178204

Descripteurs français

Pascal (Inist)
- Hérédodégénérescence spinocérébelleuse de Friedreich, Ataxie, Cytopathie mitochondriale, Pathologie du système nerveux, Essai clinique, Neurologie, Trinucléotide.

English descriptors

KwdEn :
- Ataxia, Clinical trial, Friedreich ataxia, Mitochondrial disorder, Nervous system diseases, Neurology, Trinucleotide.

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.

Notice en format standard (ISO 2709)

Pour connaître la documentation sur le format Inist Standard.

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C01	`01`		`ENG`	@0 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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Format Inist (serveur)

NO :	PASCAL 10-0178204 INIST
ET :	Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design
AU :	FRIEDMAN (Lisa S.); FARMER (Jennifer M.); PERLMAN (Susan); WILMOT (George); GOMEZ (Christopher M.); BUSHARA (Khalaf O.); MATHEWS (Katherine D.); SUBRAMONY (S. H.); ASHIZAWA (Tetsuo); BALCER (Laura J.); WILSON (Robert B.); LYNCH (David R.)
AF :	Department of Neurology, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 10 aut., 12 aut.); Department of Pediatrics, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 12 aut.); Children's Hospital of Philadelphia/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 12 aut.); Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles/Los Angeles, California/Etats-Unis (3 aut.); Department of Neurology, Emory University/Atlanta, Georgia/Etats-Unis (4 aut.); Department of Neurology, University of Minnesota/Minneapolis, Minnesota/Etats-Unis (5 aut., 6 aut.); Department of Neurology, University of Chicago/Chicago, Illinois/Etats-Unis (5 aut.); Departments of Neurology and Pediatrics, University of Iowa/Iowa City, Iowa/Etats-Unis (7 aut.); Department of Neurology, University of Texas Medical Branch/Galveston, Texas/Etats-Unis (8 aut., 9 aut.); Department of Neurology, University of Mississippi/Jackson, Mississippi/Etats-Unis (8 aut.); Department of Neurology, University of Florida/Gainesville, Florida/Etats-Unis (9 aut.); Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (11 aut.)
DT :	Publication en série; Niveau analytique
SO :	Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2010; Vol. 25; No. 4; Pp. 426-432; Bibl. 17 ref.
LA :	Anglais
EA :	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.
CC :	002B17; 002B17F
FD :	Hérédodégénérescence spinocérébelleuse de Friedreich; Ataxie; Cytopathie mitochondriale; Pathologie du système nerveux; Essai clinique; Neurologie; Trinucléotide
FG :	Pathologie de l'encéphale; Maladie dégénérative; Maladie héréditaire; Pathologie de la moelle épinière; Pathologie du système nerveux central; Trouble neurologique; Enzymopathie; Maladie métabolique
ED :	Friedreich ataxia; Ataxia; Mitochondrial disorder; Nervous system diseases; Clinical trial; Neurology; Trinucleotide
EG :	Cerebral disorder; Degenerative disease; Genetic disease; Spinal cord disease; Central nervous system disease; Neurological disorder; Enzymopathy; Metabolic diseases
SD :	Heredodegeneración espinocerebelosa Friedreich; Ataxia; Citopatía mitocondrial; Sistema nervioso patología; Ensayo clínico; Neurología; Trinucleótido
LO :	INIST-20953.354000181523490050
ID :	10-0178204

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Pascal:10-0178204

Le document en format XML

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<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>
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<s2>Iowa City, Iowa</s2>
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<s2>Philadelphia, Pennsylvania</s2>
<s3>USA</s3>
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<s2>Philadelphia, Pennsylvania</s2>
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<affiliation><inist:fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
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<sZ>1 aut.</sZ>
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<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>
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<seriesStmt><title level="j" type="main">Movement disorders</title>
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<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>
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<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>
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<fA08 i1="01" i2="1" l="ENG"><s1>Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design</s1>
</fA08>
<fA11 i1="01" i2="1"><s1>FRIEDMAN (Lisa S.)</s1>
</fA11>
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<fA11 i1="07" i2="1"><s1>MATHEWS (Katherine D.)</s1>
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<fA11 i1="10" i2="1"><s1>BALCER (Laura J.)</s1>
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<fA11 i1="12" i2="1"><s1>LYNCH (David R.)</s1>
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<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>
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<fA14 i1="02"><s1>Department of Pediatrics, School of Medicine, University of Pennsylvania</s1>
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<fA14 i1="03"><s1>Children's Hospital of Philadelphia</s1>
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<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>
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</fA14>
<fA14 i1="05"><s1>Department of Neurology, Emory University</s1>
<s2>Atlanta, Georgia</s2>
<s3>USA</s3>
<sZ>4 aut.</sZ>
</fA14>
<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>
</fA14>
<fA14 i1="07"><s1>Department of Neurology, University of Chicago</s1>
<s2>Chicago, Illinois</s2>
<s3>USA</s3>
<sZ>5 aut.</sZ>
</fA14>
<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>
</fA14>
<fA14 i1="09"><s1>Department of Neurology, University of Texas Medical Branch</s1>
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<sZ>9 aut.</sZ>
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<fA14 i1="10"><s1>Department of Neurology, University of Mississippi</s1>
<s2>Jackson, Mississippi</s2>
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<sZ>8 aut.</sZ>
</fA14>
<fA14 i1="11"><s1>Department of Neurology, University of Florida</s1>
<s2>Gainesville, Florida</s2>
<s3>USA</s3>
<sZ>9 aut.</sZ>
</fA14>
<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>
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<server><NO>PASCAL 10-0178204 INIST</NO>
<ET>Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design</ET>
<AU>FRIEDMAN (Lisa S.); FARMER (Jennifer M.); PERLMAN (Susan); WILMOT (George); GOMEZ (Christopher M.); BUSHARA (Khalaf O.); MATHEWS (Katherine D.); SUBRAMONY (S. H.); ASHIZAWA (Tetsuo); BALCER (Laura J.); WILSON (Robert B.); LYNCH (David R.)</AU>
<AF>Department of Neurology, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 10 aut., 12 aut.); Department of Pediatrics, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 12 aut.); Children's Hospital of Philadelphia/Philadelphia, Pennsylvania/Etats-Unis (1 aut., 2 aut., 12 aut.); Department of Neurology, David Geffen School of Medicine at the University of California, Los Angeles/Los Angeles, California/Etats-Unis (3 aut.); Department of Neurology, Emory University/Atlanta, Georgia/Etats-Unis (4 aut.); Department of Neurology, University of Minnesota/Minneapolis, Minnesota/Etats-Unis (5 aut., 6 aut.); Department of Neurology, University of Chicago/Chicago, Illinois/Etats-Unis (5 aut.); Departments of Neurology and Pediatrics, University of Iowa/Iowa City, Iowa/Etats-Unis (7 aut.); Department of Neurology, University of Texas Medical Branch/Galveston, Texas/Etats-Unis (8 aut., 9 aut.); Department of Neurology, University of Mississippi/Jackson, Mississippi/Etats-Unis (8 aut.); Department of Neurology, University of Florida/Gainesville, Florida/Etats-Unis (9 aut.); Department of Pathology and Laboratory Medicine, School of Medicine, University of Pennsylvania/Philadelphia, Pennsylvania/Etats-Unis (11 aut.)</AF>
<DT>Publication en série; Niveau analytique</DT>
<SO>Movement disorders; ISSN 0885-3185; Etats-Unis; Da. 2010; Vol. 25; No. 4; Pp. 426-432; Bibl. 17 ref.</SO>
<LA>Anglais</LA>
<EA>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.</EA>
<CC>002B17; 002B17F</CC>
<FD>Hérédodégénérescence spinocérébelleuse de Friedreich; Ataxie; Cytopathie mitochondriale; Pathologie du   système nerveux; Essai clinique; Neurologie; Trinucléotide</FD>
<FG>Pathologie de l'encéphale; Maladie dégénérative; Maladie héréditaire; Pathologie de la moelle épinière; Pathologie du système nerveux central; Trouble neurologique; Enzymopathie; Maladie métabolique</FG>
<ED>Friedreich ataxia; Ataxia; Mitochondrial disorder; Nervous system diseases; Clinical trial; Neurology; Trinucleotide</ED>
<EG>Cerebral disorder; Degenerative disease; Genetic disease; Spinal cord disease; Central nervous system disease; Neurological disorder; Enzymopathy; Metabolic diseases</EG>
<SD>Heredodegeneración espinocerebelosa Friedreich; Ataxia; Citopatía mitocondrial; Sistema nervioso patología; Ensayo clínico; Neurología; Trinucleótido</SD>
<LO>INIST-20953.354000181523490050</LO>
<ID>10-0178204</ID>
</server>
</inist>
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	Movement Disorders (revue)
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Movement Disorders (revue)

Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design

Measuring the Rate of Progression in Friedreich Ataxia: Implications for Clinical Trial Design

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