| Investigator | Pr T . Voit |
| Sponsor | PTC |
| CCPPRB | Pending |
| Patient numbers Total At the Institute of Myology | 165 10 |
| Projected dates Beginning Ending | June 2008 June 2010 |
| Financing | PTC |
| Outcome measures | 6 min walk; actimetry; dynamometry; biopsy; biology; PK; cognition, echoTDI |
