D.09 Pharmacological therapy for the prevention and management of cardiomyopathy in Duchenne muscular dystrophy: a systematic review

Abstract

LE JOURNAL CANADIEN DES SCIENCES NEUROLOGIQUESSuppl. 2 -S15 in Canada, with the goal of facilitating discourse that could lead to national standards for EMG training. Methods: An online survey was distributed to senior neurology and physiatry residents (postgraduate years 3-5), at seven tertiary Canadian centres. The study authors, who are trainees and consultants with a broad range of EMG expertise (junior and senior resident, clinical neuromuscular fellows, senior physiatrist and neuromuscular neurologists), developed pertinent demographic and qualitative questions. Results: Thirty-eight residents completed the survey (23 neurology, 15 physiatry). There was inter-program variation in quantity of the training experience, content of the curriculum, access to expertise (including technologists) and goals for future training and practice. Similarly, differences were identifi ed between the training experiences of neurology and physiatry residents. Conclusions: Inter-program variability in EMG training was identifi ed. Additionally, differences were identifi ed between neurology and physiatry resident training. This data provides evidence of training discrepancies across the country and can be used to establish national training standards for EMG in Canada. D.08 ACT DMD (Ataluren Confi rmatory Trial in Duchenne Muscular Dystrophy): effect of Ataluren on timed function tests (TFT) in nonsense mutation (nm) DMD N Goemans (Leuven) C Campbell (London)* CM McDonald (Sacramento) T Voit (London) X Luo (South Plainfi eld) G Elfring (South Plainfi eld) H Kroger (South Plainfi eld) P Riebling (South Plainfi eld) T Ong (South Plainfi eld) R Spiegel (South Plainfi eld) SW Peltz (South Plainfi eld) K Bushby (Newcastle upon Tyne)doi: 10.1017/cjn.2016.81Background: Ataluren is the fi rst drug to treat the underlying cause of nmDMD. Methods: ACT DMD is a Phase 3, randomized, double-blind study. Males 7-16 years with nmDMD and a screening six-minute walk distance (6MWD) ≥150m and <80%-predicted were randomized to ataluren 40 mg/kg/day or placebo for 48 weeks. A pre-specifi ed subgroup included patients with baseline 6MWD 300-400m. A meta-analysis of the overall ACT DMD population and the 'ambulatory decline phase' subgroup of the Phase 2b study (those patients meeting ACT DMD entry criteria) was pre-specifi ed in the statistical plan. Results: In the overall ACT DMD population (N=228), changes in TFTs favored ataluren over placebo: 10-meter walk/run, -1.2s (p=0.117); 4-stair climb, -1.8s (p=0.058); 4-stair descend, -1.8s (p=0.012). In the pre-specifi ed subgroup (n=99), these differences increased to -2.1s, -3.6s, and -4.3s, respectively, and were statistically signifi cant (p<0.01) for 4-stair climb and descend. Results are supported by the meta-analysis (N=291), which demonstrated significant differences (p<0.05) in 10-meter walk/run, 4-stair climb, 4-stair descend. Conclusions: TFT results showed a benefi t for ataluren in ACT DMD, and a larger treatment effect in the pre-specifi ed baseline 6MWD 300-400m subgroup as well as the...