Rare diseases

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London, UK - January 8, 2015: City of London panorama includes high street with office and governments buildings

Orchard’s gene therapy gains UK ‘promising innovative medici...

A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.

Views & Analysis

Close-up shot of an old rusty key inside a keyhole

Orphan drugs: overcoming HTA barriers to access

How can orphan drug manufacturers demonstrate value and improve access to drugs?

News

Ionis to file rare disease drug after GSK declines option

Firm turns down options for inotersen and IONIS-FB-LRX.

News

Alexion cuts ties with Moderna and re-focuses

Alexion cuts collaborations with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma

News

Promise of CF combinations lifts Vertex, reignites takeover ...

Encouraging results from three pipeline combination therapies suggests Vertex could extend treatment to patients with more severe and difficult to treat cystic fibrosis.

News

Amicus to file Fabry drug after FDA changes mind

Regulator drops demand for further phase 3 trial.

Orchard’s gene therapy gains UK ‘promising innovative medici...

Orphan drugs: overcoming HTA barriers to access

Ionis to file rare disease drug after GSK declines option

Alexion cuts ties with Moderna and re-focuses

Promise of CF combinations lifts Vertex, reignites takeover ...

Amicus to file Fabry drug after FDA changes mind

Neuralink unveils trial linking brain implant to robotic arm

The AI effect in pharma commercial strategy: From data to re...

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