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Unruffled by ‘outlier’ claims, Vertex CEO upbeat on Orkambi breakthrough

NHS England going public with price makes deal impossible, says Leiden

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‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

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Afinitor and Uptravi gain funding from NHS England

Rare disease treatments navigate tricky market access route

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Chugai gains FDA breakthrough status for rare CNS treatment

Adds to company's BTD roll-call

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Roche gains EU PRIME status for Spinraza rival

Oral treatment joins Novartis' gene therapy challenger

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NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done

Room for manoeuvre after initial rejection

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Biogen picks up another drug from wide-ranging Ionis alliance

Aims to follow success of Spinraza

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Orphan drugs from Biogen and Amicus win UK Prix Galien

New medical technology category added for the first time

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Spinraza lifts Biogen ahead of Alzheimer’s data

Continued strong growth for Biogen’s spinal muscular atrophy therapy Spinraza helped the company exceed revenue expectations in the third quarter, but pipeline disappointments kept its shares largely static.Spinraza (nusinersen) was...

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Alnylam seeks fast-track filing with biomarker data

Early filing reflects FDA desire for faster approvals

Rare diseases and orphan drugs: Our experience and expertise

We have designed a complimentary infographic outlining the market opportunity as well as our experience and expertise in the growing area of rare diseases and orphan drugs, which includes links to...

Research Partnership

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Ionis and Roche gain PRIME status for Huntingdon’s disease candidate

More good news for Ionis after first drug approval

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