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- PMLiVE

Ehlers-Danlos syndrome

Rare disease patients face 5-7 year wait for diagnosis

Doctors often lack time and resources to manage patients

Roche - Basel

Roche and Isis in $392m deal to research drugs for brain disorder

Will develop antisense drugs for Huntington's disease

- PMLiVE

Novartis provides multimedia support to Rare Diseases Day

Collaborates with Eurordis on multimedia education campaign

- PMLiVE

Shining a light on orphan medicines

Orphan diseases represent one of the most exciting and emotive areas in modern medicine and need a communications strategy that covers a spectrum of issues

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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