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XLH

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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...

- PMLiVE

Alnylam seeks fast-track filing with biomarker data

Early filing reflects FDA desire for faster approvals

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NICE U-turn on Crystiva for rare bone disease

Watchdog to back the drug to treat young people with XLH

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FDA clears Kyowa Kirin’s skin lymphoma therapy Poteligeo

Becomes the first drug approved to treat Sézary syndrome

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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Spinraza keeps Biogen buoyant ahead of Alzheimer’s data

Biotech sees 9% growth ahead of potential landmark announcement

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Sanofi’s rare blood disorder drug Cablivi set for European approval

Rare disease drug among notable CHMP recommendations

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NICE set to reject Kyowa Kirin’s Crysvita for XLH

Committee says the rare disease therapy isn’t value for money

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