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- PMLiVE

Isis Pharmacueticals

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J&J gets EU green light for novel TB drug

Sirturo approved to treat multidrug resistant form of the disease

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NICE: Alexion must justify Soliris’ high cost

UK body unable to recommend ‘ultra-orphan’ drug

Roche - Basel

Roche drug fails in lung cancer trial

Damages prospects for onartuzumab

- PMLiVE

Pharma companies back Rare Disease Day

Genzyme, Shire and more hold global campaigns today

- PMLiVE

AZ gets limited approval for metreleptin in US

First FDA-backed treatment for rare disease lipodystrophy

- PMLiVE

WCCT Global talks up orphan drug benefits of new service

CRO's In-Site programme will harness digital technology for patient recruitment

Define the patient burden in rare diseases

Putting patients and carers at the heart of the value demonstration equation in orphan drugs is key

- PMLiVE

FDA backs BioMarin drug for rare enzyme disorder

Vimizim is first drug approved in US under rare paediatric disease priority review

- PMLiVE

EMA to expand orphan drug HTA collaboration

Will further develop the concept of parallel scientific advice in 2014

- PMLiVE

Orphan drugs: A ‘responsible’ investment

There's growing sense of responsibility in the orphan drug market, but the market remains under-exploited by companies

National Institute for Health and Care Excellence NICE logo

NICE reviews lung cancer drug guidance

Consults on availability of Roche's Tarceva and AZ's Iressa on NHS

- PMLiVE

Supportive strategies for demonstrating value

Improved planning will support better data generation and improve understanding of rare diseases and the orphan drugs that treat them

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