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- PMLiVE

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- PMLiVE

FDA gives Alexion’s metabolic drug breakthrough status

Reduces requirements for registration, potentially speeding up approval

Healthcare at Home and QED partner on orphan diseases

OrphanReach alliance will see companies provide product lifecycle services

Payer focus in the personalised world of oncology and orphan diseases

Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the new scientific understanding

Novartis building

Novartis gets FDA green light for new Ilaris indication

Immunotherapeutic wins US approval for severe form of childhood arthritis

- PMLiVE

FDA approves drugs for rare genetic liver condition and to reverse anticoagulation

Raptor’s Procysbi and CSL Behring’s Kcentra recommended in US

- PMLiVE

European drug applications ‘stable but more complex in 2012’

Despite austerity measures, EMA's annual report shows signs of biopharma resilience

Rare disease patients face 5-7 year wait for diagnosis

Doctors often lack time and resources to manage patients

Roche - Basel

Roche and Isis in $392m deal to research drugs for brain disorder

Will develop antisense drugs for Huntington's disease

- PMLiVE

Otsuka files new TB candidate in Japan as shortages worsen

Delamanid shows promise in multi-drug resistant TB but supplies of rifampin are low

- PMLiVE

Takeda files lymphoma drug brentuximab vedotin in Japan

Seeks approval for antibody-drug conjugate for two forms of the blood cancer

- PMLiVE

Novartis provides multimedia support to Rare Diseases Day

Collaborates with Eurordis on multimedia education campaign

- PMLiVE

Shining a light on orphan medicines

Orphan diseases represent one of the most exciting and emotive areas in modern medicine and need a communications strategy that covers a spectrum of issues

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