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- PMLiVE

Rare Disease Day 2021

- PMLiVE

Baxalta agrees to $32bn Shire takeover

Deal will  create one of the industry's top rare disease firms

- PMLiVE

Another lost decade for research?

What does the patent landscape look like for antibiotics, vaccines and orphan drugs?

- PMLiVE

Mixed news for gene therapies at ASH conference

GSK unveils successful trial, but there was disappointing news for Bluebird

EU flag

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

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