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- PMLiVE

Spectrum Pharmaceuticals

- PMLiVE

EU and Australian regulators collaborate on rare diseases

EMA and Therapeutic Goods Administration to share reports for orphan drugs

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Europe votes for greater clinical trial transparency

European Parliament passes new EU Clinical Trials Regulation

- PMLiVE

Final NICE backing for MabThera in rare condition

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

- 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’

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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

- 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

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

- PMLiVE

FDA approves Chelsea’s rare blood pressure drug

US biopharma company gets green light in US for Northera

Define the patient burden in rare diseases

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

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