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- PMLiVE

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Wakix backed for European approval in narcolepsy

Bioprojet Pharma’s orphan drug given a positive opinion by CHMP

- PMLiVE

Orphan drug firm Sobi opens new European base

Company'sBrussels office will also house its Benelux operations

- PMLiVE

FDA grants Tocagen’s glioblastoma drug orphan status

Puts the brain cancer gene therapy in-line for development incentives

- PMLiVE

Shire launches rare disease website

Hereditary angioedema sitesupports its Me, Not HAE campaign

Shire Basingstoke

Shire links with US hospital for rare disease research

Willwork with Cincinnati Children's Hospital Medical Centre novel therapies

- PMLiVE

FDA grants orphan drug status to Treeway’s ALS treatment

Amyotrophic Lateral Sclerosis drug TW001 gainsregulatory benefits

- PMLiVE

NICE backs first ‘ultra-orphan’ drug Soliris

Final guidance backs Alexion’s treatment for very rare blood clot condition

- PMLiVE

Boehringer drug approved in EU for rare lung disease

Nintedanib is already available as cancer treatment

Shire Basingstoke

Shire signs deal to buy NPS for $5.2bn

Firm hopes to boost its presence in rare disease sector

- PMLiVE

Assessing drugs for ultra-rare conditions in the UK

Alexion’s Soliris has become the first drug to pass through NICE’s new highly specialised technology process

- PMLiVE

Cancer drugs lead SMC recommendations

New treatments from Roche, Lilly and Celgene backed for use in Scotland

- PMLiVE

SMC fails to recommend Roche’s Perjeta

Says drug is not a cost-effective option in breast cancer

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