'Historic' first ever drug for Duchenne muscular dystrophy approved - Telegraph: The first ever drug to combat Duchenne muscular dystrophy, a life threatening muscle wasting condition, has been approved by medicines regulators and hailed by experts as 'historic'.
Translarna, also known as ataluren, prompts the body to manufacture a protein that protects against muscle damage which is absent in children with the disease.
The drug has been licensed under special measures allowed for medicines to treat life threatening diseases where no other alternative drug is available.
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