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

FDA clears first therapy for rare childhood cancer

United Therapeutics’ Unituxin approved for neuroblastoma

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Jinarc backed for European approval in rare kidney disease

Positive opinion for Otsuka comes just before rare disease awareness day

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Europe gains access to first stem cell drug

Chiesi's Holoclar has been given the green light for a rare eye condition

- PMLiVE

Imbruvica gets FDA OK for rare lymphoma

Cancer drug available in US to treat Waldenström's macroglobulinaemia          

- PMLiVE

NICE backs first ‘ultra-orphan’ drug Soliris

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

- 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

FDA approves Ipsen drug to treat rare tumours

Green light for Somatuline Depot in cancer of the neuroendocrine system

- PMLiVE

Celgene wins orphan drug award at PMEA

Company is recognised for providing early access to Imnovid in multiple myeloma

- 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

EMA offers orphan drug benefits for Ebola research

Incentives to encourage development of new treatments for the virus

- PMLiVE

Janssen’s blood cancer drug Imbruvica approved in Europe

European commission licenses the first-in-class drug for two rare diseases