Log in with your email address username.

×

Attention doctorportal newsletter subscribers,

After December 2018, we will be moving elements from the doctorportal newsletter to MJA InSight newsletter and rebranding it to Insight+. If you’d like to continue to receive a newsletter covering the latest on research and perspectives in the medical industry, please subscribe to the Insight+ newsletter here.

As of January 2019, we will no longer be sending out the doctorportal email newsletter. The final issue of this newsletter will be distributed on 13 December 2018. Articles from this issue will be available to view online until 31 December 2018.

[Articles] Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Change in 6MWD did not differ significantly between patients in the ataluren group and those in the placebo group, neither in the intention-to-treat population nor in the prespecified subgroups with a baseline 6MWD of less than 300 m or 400 m or more. However, we recorded a significant effect of ataluren in the prespecified subgroup of patients with a baseline 6MWD of 300 m or more to less than 400 m. Baseline 6MWD values within this range were associated with a more predictable rate of decline over 1 year; this finding has implications for the design of future DMD trials with the 6-minute walk test as the endpoint.

email