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- PMLiVE

European Court of Justice

Sanofi reception

US approval for Sanofi’s Kynamro in genetic cholesterol disorder

Comes one month after drug was turned down in Europe due to safety concerns

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

Scotland launches £21m fund for rare conditions

Will pay for orphan medicines not recommended by the SMC

- PMLiVE

PatientsLikeMe takes aim at one of world’s rarest diseases

Creates open registry and community for alkaptonuria patients

- PMLiVE

FDA clears six new drugs in time for the new year

Brings 2012 tally to 39 – the highest for more than 15 years

- PMLiVE

Year-end FDA approvals for Novartis, GSK drugs

Agency gives go-ahead to Cushing’s disease treatment Signifor and raxibacumab for inhalational anthrax

Genzyme enters $22.5m RNAi deal with Alnylam in Asia

Programme targets condition affecting heart and nervous system

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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