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- PMLiVE

EURORDIS

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FDA sets up rare disease research fund

Will invest $14m across 15 orphan drug projects

Orphan drugs: the early access regulatory environment

Despite considerable variation to the rules for unlicensed orphan drugs, there are some clear first steps for a global access programme

- PMLiVE

Intermune supports awareness efforts for IPF

Last week's IPF World Week saw the global patient community unite

- 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

Shire extends rare genetic disease alliance

Agrees option with Santaris to nominate additional discovery targets

- PMLiVE

Novartis orphan drug wins ‘breakthrough’ status from FDA

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

EU flag

EC sets up expert group on rare diseases

Will revamp EUCERD to better handle development of orphan medicines

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