| Title |
Friedreich Ataxia Clinical Outcome Measures
|
|---|---|
| Published in |
Journal of Child Neurology, June 2012
|
| DOI | 10.1177/0883073812448462 |
| Pubmed ID | |
| Authors |
Sean R. Regner, Nicholas Wilcox, Lisa S. Friedman, Lauren Seyer, Kim Schadt, Karlla W. Brigatti, Susan Perlman, Martin Delatycki, George R. Wilmot, Christopher M. Gomez, Khalaf O. Bushara, Katherine D. Mathews, S.H. Subramony, Tetsuo Ashizawa, Bernard Ravina, Alicia Brocht, Jennifer M. Farmer, David R. Lynch |
| Abstract |
Friedreich ataxia is an autosomal recessive neurodegenerative disorder characterized by ataxia, dysarthria, and areflexia. The authors report the progress of a large international noninterventional cohort (n = 410), tracking the natural history of disease progression using the neurologic examination-based Friedreich Ataxia Rating Scale. The authors analyzed the rate of progression with cross-sectional analysis and longitudinal analysis over a 2-year period. The Friedreich Ataxia Rating Scale captured disease progression when used at 1 and 2 years following initial evaluation, with a lower ratio of standard deviation of change to mean change over 2 years of evaluation. However, modeling of disease progression identified substantial ceiling effects in the Friedreich Ataxia Rating Scale, suggesting this measure is most useful in subjects before maximal deficit is approached. |
Login to access the Attention Digest and the Sentiment Analysis related to this output.
X Demographics
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United Kingdom | 1 | 50% |
| Unknown | 1 | 50% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 50% |
| Scientists | 1 | 50% |
Mendeley demographics
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 1 | 1% |
| Chile | 1 | 1% |
| Unknown | 71 | 97% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 14 | 19% |
| Other | 10 | 14% |
| Student > Master | 8 | 11% |
| Student > Bachelor | 5 | 7% |
| Professor | 5 | 7% |
| Other | 15 | 21% |
| Unknown | 16 | 22% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 19 | 26% |
| Agricultural and Biological Sciences | 7 | 10% |
| Neuroscience | 7 | 10% |
| Biochemistry, Genetics and Molecular Biology | 4 | 5% |
| Pharmacology, Toxicology and Pharmaceutical Science | 3 | 4% |
| Other | 13 | 18% |
| Unknown | 20 | 27% |