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Analysis

Lonafarnib, a hope for progeria treatment

Lonafarnib has emerged as a potential treatment for progeria and progeroid laminopathies, an ultra-rare condition for which no approved treatment exists. Having successfully demonstrated survival benefits in progeria patients, lonafarnib is improving the hope for the patients, who generally succumb to death at an early age of 14 years. The drug currently has orphan drug, breakthrough therapy, as well as rare paediatric disease (RPD) designations from the US Food and Drug Administration (FDA).

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