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- PMLiVE

Raptor Pharmaceutical

- PMLiVE

Personalised understanding: oncology and orphan diseases

Improvements in our understanding of cancer are drawing the orphan drug and oncology worlds ever closer together

- PMLiVE

J&J files rare blood disorder drug siltuximab in US and EU

Orphan drug to be assessed for use in the treatment of multicentric Castleman disease

Novartis building

Novartis wins childhood arthritis indication for Ilaris in EU

EC gives green light to drug for the treatment of systemic juvenile idiopathic arthritis

Novartis building

NICE backs Novartis’ Jetrea in rare eye condition

Recommends drug be available for NHS use to treat vitreomacular traction

- PMLiVE

ReNeuron wins orphan status for stem cell therapy

ReN003 is being assessed as a treatment for rare eye disorder retinitis pigmentosa

- PMLiVE

Chiesi acquires Zymenex and mulls new rare disease unit

Deal adds late-stage orphan drug to Italian pharma company's pipeline

- PMLiVE

Novartis orphan drug wins ‘breakthrough’ status from FDA

Bimagrumab could be first treatment for rare life-threatening muscle-wasting condition

- PMLiVE

FDA panel turns down Otsuka’s drug for rare kidney disease

Unable to recommend expanded use of tolvaptan

EU flag

EC sets up expert group on rare diseases

Will revamp EUCERD to better handle development of orphan medicines

- PMLiVE

Boehringer: clinical trial set-up poses challenge in rare diseases

Says more support needed in regulatory process for orphan drugs

Actelion HQ Switzerland

Actelion to buy Ceptaris – if FDA backs its cancer treatment

Acquisition contingent on Valchlor approval in rare lymphoma

BTG extends orphan drug distribution partnership with Clinigen

Deal covers treatment for overexposure to chemotherapy

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