Queen Square Centre for Neuromuscular Diseases


A study of PRO053 in subjects with Duchenne muscular dystrophy (DMD)

Sponsor Prosensa
CI Professor Volker Straub
sites Newcastle and London GOSH

Newcastle: becky.davis@ncl.ac.uk

London: c.uzowuru@ucl.ac.uk

More information

Primary objective

To determine the benefit of treatment by assessing the six minute walking distance (6MWD) test after 48 weeks on the selected dose of the drug.


42 male patients with DMD resulting from an exon deletion

6 out of 42 will participate in the dose-escalation and 48-week treatment phases, and 36 will only participate in the 48-week treatment phase.

To be evaluable, patients must be able to walk at least 230 metres in 6 minutes at the start of the treatment phase once the optimum dose has been selected.