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- PMLiVE

EURORDIS

Lonafarnib: promising results in first-ever trial for progeria

Treatments for progeria are limited but lonafarnib and FTIs as a class have given renewed hope for children with the condition

- PMLiVE

PatientsLikeMe takes aim at one of world’s rarest diseases

Creates open registry and community for alkaptonuria patients

European orphan drug applications up 30 per cent

But lower than expected generics applications see EMA reduce new marketing authorisation forecast

- PMLiVE

Orphan drugs economically viable for pharma

Drugs for rare diseases can generate as much lifetime revenue as medicines for common conditions

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