Journal article
Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
Movement disorders, Vol.25(4), pp.426-432
03/15/2010
DOI: 10.1002/mds.22912
PMCID: PMC2954653
PMID: 20063431
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. The present 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 two years than over one 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.
Details
- Title: Subtitle
- Measuring the rate of progression in Friedreich ataxia: Implications for clinical trial design
- Creators
- Lisa S Friedman - Department of Neurology, University of Pennsylvania School of Medicine, United StatesJennifer M Farmer - Department of Neurology, University of Pennsylvania School of Medicine, United StatesSusan Perlman - University of California Los Angeles, Los Angeles, CA, United StatesGeorge Wilmot - Emory University, Atlanta, GA, United StatesChristopher Gomez - University of Minnesota, Minneapolis, MN, United StatesKhalaf O Bushara - University of Minnesota, Minneapolis, MN, United StatesKatherine D Mathews - University of Iowa, Iowa City, IA, United StatesS. H Subramony - University of Texas Medical Branch, Galveston, TX, United StatesTetsuo Ashizawa - University of Texas Medical Branch, Galveston, TX, United StatesLaura J Balcer - Department of Neurology, University of Pennsylvania School of Medicine, United StatesRobert B Wilson - Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, United StatesDavid R Lynch - Department of Neurology, University of Pennsylvania School of Medicine, United States
- Resource Type
- Journal article
- Publication Details
- Movement disorders, Vol.25(4), pp.426-432
- DOI
- 10.1002/mds.22912
- PMID
- 20063431
- PMCID
- PMC2954653
- NLM abbreviation
- Mov Disord
- ISSN
- 0885-3185
- eISSN
- 1531-8257
- Grant note
- DOI: 10.13039/100002108, name: Friedreich Ataxia Research Alliance; DOI: 10.13039/100005202, name: Muscular Dystrophy Association
- Language
- English
- Date published
- 03/15/2010
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984020898302771
Metrics
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