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Muscular Dystrophy

Question for Department of Health

UIN 200116, tabled on 10 June 2014

To ask the Secretary of State for Health, what progress has been made in licensing drugs to deal with Duchenne muscular dystrophy.

Answered on

17 June 2014

On 22 May 2014, the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended granting a conditional marketing authorisation for Translarna (ataluren), an orphan designated medicine for the treatment of Duchenne muscular dystrophy caused by nonsense mutations. Translarna is to be used in patients aged five years and older who are able to walk.

The CHMP opinion on Translarna will now be sent to the European Commission for adoption of a decision on an European Union-wide marketing authorisation.

Other products are currently in various stages of development.