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Muscular Dystrophy: Drugs

Volume 461: debated on Friday 15 June 2007

To ask the Secretary of State for Health (1) what plans her Department has to fast-track new drugs and therapies for Duchenne muscular dystrophy; (141446)

(2) if she will make the drug PTC-124 available through the NHS to all boys with Duchenne muscular dystrophy with single point variations of the gene;

(3) what steps her Department has taken to identify boys with Duchenne muscular dystrophy with single point variations of the gene.

We have no specific plans to fast-track new drugs or therapies for the treatment of Duchenne muscular dystrophy.

PTC124 is a drug currently undergoing clinical trials in the United States for the treatment of Duchenne muscular dystrophy. PTC Therapeutics, the company behind this drug, has not currently organised clinical trials in the United Kingdom.

We have not taken any specific steps to identify those boys with Duchenne muscular dystrophy who would benefit from the drug PTC124; those whose disease is caused by the ‘nonsense’ mutation.