| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 10833478 | Molecular Genetics and Metabolism | 2012 | 6 Pages |
Abstract
⺠We performed a prospective open label trial of the novel therapeutic EPI-743 for children with Leigh syndrome ⺠There was a statistically significant improvement in all trial outcome measurements signifying arrest and reversal of disease progression ⺠There were no drug related adverse events or clinical laboratory abnormalities
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Authors
Diego Martinelli, Michela Catteruccia, Fiorella Piemonte, Anna Pastore, Giulia Tozzi, Carlo Dionisi-Vici, Giuseppe Pontrelli, Tiziana Corsetti, Susanna Livadiotti, Viktoria Kheifets, Andrew Hinman, William D. Shrader, Martin Thoolen, Matthew B. Klein,