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- PMLiVE

orphen drugs

- PMLiVE

NICE demands more data from Roche for MabThera in rare autoimmune disease

Unable to recommend expanding drug’s indication to include vasculitis

Enabling access for patients with rare diseases

When it comes to meeting patient needs, the well-known counsel to ‘think globally, act locally’ rings true

EC consults on orphan drug application guidelines

Also seeks input on the transfer of designations from one sponsor to another

- PMLiVE

Analysts question value of rumoured $20bn Roche bid for Alexion

Shares in orphan drug specialist slide back after earlier surge

- PMLiVE

Court orders EC to approve rejected orphan medicine

Says CTRS should be allowed to market Orphacol for the treatment of rare bile condition

- PMLiVE

Revlimid faces NICE rejection for use in rare blood cancer

Watchdog's draft guidance does not recommend Celgene's drug for NHS use in England and Wales

Novartis sponsors rare disease patient community

Backs site for patients affected by pancreatic neuroendocrine tumours (PNET)

- PMLiVE

Rare diseases among initial priorities of UK DNA mapping body

Genomics England will also help advance research into cancer and infectious diseases

- PMLiVE

FDA grants orphan status to Activartis’ brain cancer immunotherapy

Regulator says AV0113 has potential in rare cancer malignant glioma

- PMLiVE

Alexion begins work on new global headquarters

Rare diseases drug company will move to new US site in 2015

- PMLiVE

FDA gives priority review status to BMS/ AZ’s metreleptin

Former obesity prospect has potential in the rare disease lipodystrophy

- PMLiVE

Orphan status in US for Astellas/ Basilea antifungal

Isavuconazole is being developed as a treatment for invasive aspergillosis

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