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- PMLiVE

Acer Therapeutics

EphMRA 2017 round up

We were delighted to present, exhibit and sponsor this year’s EphMRA Annual Conference in Amsterdam, Netherlands.

Research Partnership

- PMLiVE

Focus on Rare Diseases

As pharma becomes more focused on rare disease therapies, Lucid Group recognises that medical education agencies need to respond.

Lucid Group Communications Limited

Shire Basingstoke

Shire finally bags EU approval for rare disease drug Natpar

Becomes first licenced treatment for hypoparathyroidism in Europe

Blending medical and digital for effective rare disease campaigns

The fundamentals of building a collaborative medical and digital team

National Institute for Health and Care Excellence NICE logo

Amicus gets NICE green light for Fabry drug

Oral therapy alternative Galafold approved for routine NHS use

- PMLiVE

NICE blocks second Alexion rare disease drug in a week

Says high cost and “considerable uncertainties” over long-term benefits prohibit routine NHS use

ABPI London offices

UK pharma slams new drug evaluation plans

ABPI and BIA oppose "arbitrary" proposal to impose £100,000 QALY limit on rare disease therapies

ABPI London offices

UK pharma slams new drug evaluation plans

ABPI and BIA oppose "arbitrary" proposal to impose £100,000 QALY limit on rare disease therapies

- PMLiVE

A rare talent

Shire’s Sebastian Stachowiak on dealing with change and working for a fit-for-purpose orphan drug environment

Biogen Idec building

FDA approves Biogen’s Spinraza for spinal muscular atrophy

Analysts predict blockbuster sales as drug is priced at $750,000 for first year of treatment

- PMLiVE

Intercept gets EU nod for rare liver disease therapy

Ocaliva approved as a combination treatment for primary biliary cholangitis

- PMLiVE

Shire to focus rare disease R&D with new US facility

Cambridge, Massachusetts hub to drive firm’s push to become leading rare disease specialist

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