| Title |
SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy
|
|---|---|
| Published in |
PLOS ONE, July 2011
|
| DOI | 10.1371/journal.pone.0021296 |
| Pubmed ID | |
| Authors |
John T. Kissel, Charles B. Scott, Sandra P. Reyna, Thomas O. Crawford, Louise R. Simard, Kristin J. Krosschell, Gyula Acsadi, Bakri Elsheik, Mary K. Schroth, Guy D'Anjou, Bernard LaSalle, Thomas W. Prior, Susan Sorenson, Jo Anne Maczulski, Mark B. Bromberg, Gary M. Chan, Kathryn J. Swoboda |
| Abstract |
Multiple lines of evidence have suggested that valproic acid (VPA) might benefit patients with spinal muscular atrophy (SMA). The SMA CARNIVAL TRIAL was a two part prospective trial to evaluate oral VPA and L-carnitine in SMA children. Part 1 targeted non-ambulatory children ages 2-8 in a 12 month cross over design. We report here Part 2, a twelve month prospective, open-label trial of VPA and L-carnitine in ambulatory SMA children. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| United States | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 154 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| Germany | 1 | <1% |
| France | 1 | <1% |
| United Kingdom | 1 | <1% |
| Spain | 1 | <1% |
| United States | 1 | <1% |
| Unknown | 149 | 97% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Student > Master | 25 | 16% |
| Student > Ph. D. Student | 22 | 14% |
| Student > Bachelor | 22 | 14% |
| Researcher | 18 | 12% |
| Student > Postgraduate | 11 | 7% |
| Other | 19 | 12% |
| Unknown | 37 | 24% |
| Readers by discipline | Count | As % |
|---|---|---|
| Medicine and Dentistry | 32 | 21% |
| Biochemistry, Genetics and Molecular Biology | 18 | 12% |
| Neuroscience | 16 | 10% |
| Agricultural and Biological Sciences | 13 | 8% |
| Nursing and Health Professions | 11 | 7% |
| Other | 24 | 16% |
| Unknown | 40 | 26% |