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- PMLiVE

EURORDIS

- 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

Extra stomach cancer indication for Cyramza in US

FDA backs Lilly drug in combination with paclitaxel chemotherapy

- PMLiVE

Vertex submits Kalydeco combo for cystic fibrosis

Seeks approval in both US and Europe

- PMLiVE

Orphan drug market to reach $176bn by 2020

New report suggests market will almost double in five years

- PMLiVE

FDA approves Baxter drug for rare form of haemophilia

Obizur is derived from pig proteins

- PMLiVE

Orphan drugs lead CHMP recommendations

Clinuvel's Scenesse is first drug set for European approval to treat rare intolerance to sunlight

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