Muscular Dystrophy: Drugs

(asked on 10th December 2014) - View Source

Question to the Department of Health and Social Care:

To ask Her Majesty’s Government why they have withdrawn approval of the drug Translarna, introduced for the treatment of Duchenne muscular dystrophy, despite the preliminary results achieved in some patients in trials in Newcastle upon Tyne.


Answered by
Earl Howe Portrait
Earl Howe
Deputy Leader of the House of Lords
This question was answered on 23rd December 2014

The Government has not withdrawn any approvals for Translarna (ataluren).

We understand that a draft clinical commissioning policy to cover the use of Translarna for the treatment of Duchenne muscular dystrophy is currently going through NHS England's standard development and decision making processes.

NHS England remains committed to ensuring that a timely decision is made about this treatment as one of a range of investment proposals that has been put forward for the 2015-16 financial year.

NHS England continues to keep the range of services and treatments available to patients under review as new evidence and expert guidance becomes available.

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