number of DMD boys assessed and treated according to standardised protocols. The support of Muscular Dystrophy Campaign for the NorthStar Clinical Network (full list of participating centers http:// www.muscular-dystrophy.org/assets/0001/5872/NSCN_collaborating_ centres.pdf) is gratefully acknowledged. doi:10.1016/j.nmd.2011.06.970 P4.6 Efficacy of low-dose steroid therapy for scoliosis in patients with Duchenne muscular dystrophy T. Murakami , K. Ishigaki, T. Saito, T. Sato, S. Onai, M. Osawa Tokyo Women’s Medical University, Pediatrics, Tokyo, Japan Since the effects of steroid therapy were reported for prolonging ambulation, cardiac and respiratory functions and preventing the pro- gression of scoliosis in patients with Duchenne muscular dystrophy (DMD), long-term oral medication is currently the mainstay of steroid therapy for DMD. In this study, we evaluated the effect of low-dose and long-term steroid therapy on scoliosis in patients with DMD. In non-ambulatory DMD patients, the severity of scoliosis was evaluated in steroid-treated and non-steroid-treated groups. Twenty three DMD patients were evaluated. Steroids were not used in 12 patients (age 9– 31 years: mean 17.72 years) and were used in the other 11 (age 10– 24 years: mean 18.18 years). The steroid therapy regimen was 0.5 mg/ kg/day of prednisolone every other day, and the mean duration of ther- apy was 9.06 years (1–18 years). The mean age of losing ambulation was 11.0 years in the steroid-treated group and 10.0 years in non-steroid-trea- ted group, without a statistically difference. In patients more than 20 years of age, Cobb° angle was over 100° in 3 of 4 patients with a mean of 80.5° (5–115°) in the non-steroid-treated group, while it exceeded 100° in only 1 of 6 patients with a mean of 42.8° (2–100°) in the steroid-treated group. Our results clearly indicate slowing of the progression of scoliosis and reduced severity even in the low-dose steroid-treated group. There was no difference in the age of losing ambulation between two groups, suggesting not only locomotor function but also other factors such as maintenance of trunk muscle strength to be involved in the prevention of scoliosis. doi:10.1016/j.nmd.2011.06.971 P4.7 How bad is deflazacort for bones in boys with Duchenne Muscular Dystrophy? A.L. Mayo a , L. McAdam b , W.D. Biggar b , B.C. Craven c a University of Toronto, Physiatry, Toronto, Canada; b University of Toronto, Holland Bloorview Kids Rehab, Pediatrics, Toronto, Canada; c University of Toronto, Toronto Rehab Institute, Physiatry, Toronto, Canada Quality of life for boys with Duchenne Muscular Dystrophy (DMD) has improved significantly with corticosteroid treatment despite its neg- ative skeletal effects. We report changes in bone health in boys with DMD on long-term deflazacort. Thirty-eight boys were evaluated pro- spectively: prior to deflazacort (T0) (0.9 mg/kg/day, maximum 39 mg/ day) and then subsequently for 1–8 years. Calcium and vitamin D were recommended. Bone health was defined by lumbar spine (L1–4) DXA Z-score (Hologic4500A, Waltham MA) at T0 then every 1–2 years, and fractures. Boys were examined quarterly. Boys with back pain had a spine X-ray. At T0, 38 ambulating boys, aged (mean ± SD) 6.7 ± 1.7 years, had Z-score À1.7 ± 0.7. By 1.7 ± 0.5 years after T0, 35 of 37 boys, aged 8.4 ± 1.7 years, had Z-score À2.1 ± 0.8. By 3.5 ± 0.6 years after T0, 23 of 33 boys, aged 10.6 ± 0.6 years, had Z-score À2.5 ± 0.8. By 5.1 ± 0.6 yearrs after T0, 19 of 23 boys aged 12.0 ± 1.9 years, had Z-score À2.9 ± 0.7. Finally by 7.2 ± 0.7 years after T0, 13 of 19 boys aged 14.2 ± 1.9 years, had Z-score À3.8 ± 0.7. Of these 13, eight became non-ambulatory, Z-score À4.3 ± 0.4; five contin- ued ambulating, Z-score À3.1 ± 0.6. Two boys had long-bone fractures before T0 and 6 boys 4.0 ± 2.3 years after T0 aged 10.3 ± 3.1 years, Z-score À2.7 ± 0.9. Four boys, aged 13.3 ± 1.7 years, had vertebral fractures 6.4 ± 0.7 years after T0, Z-score À3.7 ± 0.7. Most boys had low bone mass for age at T0. Z-scores decreased in ambulatory boys on deflazacort by approximately 0.25/years. Boys met Z-score criteria for osteoporosis (<À2.0) 4 years after T0. Non-ambulatory boys had lower Z-scores than ambulatory boys after similar years of deflazacort. Deflazacort does not appear to increase the risk of long-bone fractures compared to historical, non-treated, controls. However, treated boys have an increased incidence of vertebral fractures. As corticosteroid treatment of DMD will continue, there is an urgent need for improved management of bone health. doi:10.1016/j.nmd.2011.06.972 P4.8 Results from a two-year open label intervention study with idebenone (Catena Ò ) in Duchenne muscular dystrophy G.M. Buyse a , D. Thijs b , N. Goemans c , M. van den Hauwe c , H. Wei d , C. Rummey e , T. Meier e , L. Mertens d a University Hospitals Leuven, Child Neurology, Leuven, Belgium; b Uni- versity Hospitals Leuven, Pediatric Cardiology, Leuven, Belgium; c Univer- sity Hospitals Leuven, Pediatric Neurology, Leuven, Belgium; d The Hospital for Sick Children, Cardiology, Toronto, Canada; e Santhera Pharmaceuticals, Liestal, Switzerland A 12-month double-blind, randomized, placebo-controlled phase II study in 21 patients with DMD (DELPHI study) has indicated that idebenone (Catena Ò ) may improve cardiac and respiratory function in 8–16 year old patients with DMD. Patients completing the DELPHI study were eligible to enroll into the open label extension study (DELPHI-E) of 24 months duration to further investigate the long-term safety and efficacy of idebenone. Patients were enrolled into DELPHI-E and received Catena Ò at 450 mg/d (patients 6 45 kg body weight) or 900 mg/d (patients >45 kg body weight). Efficacy was assessed as change from baseline to month 6, 12, 18 and 24 for respiratory function tests (including peak expi- ratory flow (PEF), forced vital capacity (FVC), maximum mouth pres- sures). Cardiac function tests were conducted by echocardiography and Tissue Doppler Myocardial Imaging, particularly by assessment of the peak systolic radial strain of the left ventricular inferolateral wall, the region of the heart that is the earliest and most severely affected in DMD. Nineteen patients at age 15.1 ± 2.6 years (mean ± SD) were enrolled and completed the 24 months study period of DELPHI-E. Eleven patients were taking concomitant glucocorticoids. Catena Ò was safe and generally well tolerated. Baseline respiratory function data for the entire study cohort were: FVC: 2.20 ± 0.85 l, FVC percent predicted: 68.8 ± 25.6%, PEF: 269.4 ± 93.6 l/min, PEF percent predicted: 66.1 ± 22.3%. The change over time in respiratory as well as cardiac function data will be presented. Data from this long-term open label study will inform on the usefulness of different respiratory and cardiac efficacy outcome mea- sures for intervention trials in DMD. The study also will provide addi- tional data to assess the safety and therapeutic potential of idebenone (Catena Ò ) in patients with DMD. Study sponsored by Santhera Pharmaceuticals. doi:10.1016/j.nmd.2011.06.973 706 Abstracts / Neuromuscular Disorders 21 (2011) 639–751