Simple powder to beat 2400 genetic diseases

A powdered drug which eases the symptoms of incurable genetic diseases could be on sale within two years, researchers say.

They hope it could treat 2,400 conditions, including some types of cystic fibrosis and Duchenne muscular dystrophy, as well as the blood clotting disease haemophilia.

Many inherited diseases are caused by mutations in genes which stop cells from making vital proteins.

The new drug – known only as PTC124 – makes cells less sensitive to these mutations.

Given early enough, PTC124 could even halt the progress of some genetic illnesses – many of which are incurable.

The drug comes as a vanilla-flavoured powder to make it easier for patients to take.

This would be dissolved in water, milk or juice and taken with meals.

Researcher Dr Stuart Peltz said that there were many serious inherited diseases for which there is currently no effective treatment.

‘We hope to be able to bring this to market not only quickly for cystic fibrosis but over time to many different patients with many different diseases for which there is no treatment,’ he added.


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