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- PMLiVE

Lumena Pharmaceuticals

- PMLiVE

BioAlliance and Topotarget merge to focus on rare cancers

French and Danish firms combine to develop orphan oncology drugs

- PMLiVE

EU and Australian regulators collaborate on rare diseases

EMA and Therapeutic Goods Administration to share reports for orphan drugs

- PMLiVE

Final NICE backing for MabThera in rare condition

Roche drug available in England and Wales to treat rare form of vasculitis

- PMLiVE

Amgen cholesterol antibody shines in rare disease trial

Evolocumab passes late-stage study

- PMLiVE

No price ceiling for rare disease drugs, say MPs

BIA survey comes ahead of consultation on evaluation framework for ultra-orphans

NICE and ‘ultra orphans’

NICE and its processes for ‘highly specialised therapies’

EU flag

J&J gets EU green light for novel TB drug

Sirturo approved to treat multidrug resistant form of the disease

National Institute for Health and Care Excellence NICE logo

NICE: Alexion must justify Soliris’ high cost

UK body unable to recommend ‘ultra-orphan’ drug

- 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

The distressing impact of rare diseases

Much more needs to be done to address the problem of effective communications with stakeholders and adequate information for patients affected

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