PARLIAMENTARY EARLY DAY MOTION
ACCESS TO DUCHENNE MUSCULAR DYSTROPHY TREATMENT, TRANSLARNA (18 October 2015)

That this House notes the draft guidance published by NICE to not approve the Duchenne muscular dystrophy treatment called Translarna at this stage; acknowledges that NICE has requested further data on cost and information from the latest phase of the clinical trial from PTC Therapeutics, the pharmaceutical company which manufactures the drug; recognises that a boy in Scotland affected by Duchenne muscular dystrophy will shortly receive Translarna after an individual funding request to the local NHS Board; expresses concern from Muscular Dystrophy UK and the Duchenne community that families whose children could benefit from the treatment have waited over a year since European Commission approval for Translarna; further recognises that boys can only take the drug if they are aged five and over and still walking; further notes that Translarna is available to patients in several European countries; and calls on NICE to urgently reach a positive decision at the next stage of the process.

Sponsored by:
Mary Glindon (Labour)