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- PMLiVE

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- 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

Deal Watch March 2017

Sanofi, MedImmune, AstraZeneca, Astellas, Novartis and more feature in this month's pharma deals round-up

Blending medical and digital for effective rare disease campaigns

The fundamentals of building a collaborative medical and digital team

- PMLiVE

Astellas agrees €800m deal to buy Belgian biotech Ogeda

Will pay another €300m if fezolinetant meets clinical and regulatory targets

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

- PMLiVE

Astellas names Linda Friedman as EVP, general counsel

She is due to take up the role on 1 April

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

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