Rare diseases

News

Regeneron preps for 2020 filing for new cholesterol drug aft...

Regeneron has announced data that could create a niche for its latest cholesterol drug evinacumab in a small group of patients who do not respond to the strongest available medicines.

News

Novartis axed Avexis execs ahead of Zolgensma scandal

The plot thickens at Novartis, which is becoming embroiled in a scandal over falsification of data related to its rare disease gene therapy Zolgensma.

News

Pharming buys rare disease drug from Novartis for $20m

Pharming has bought a late-stage pipeline drug aimed at a rare disease of the immune system from Novartis for $20 million up front, plus other payments further down the line.

News

Sanofi ups revenues forecasts as eczema drug Dupixent surges

Sanofi has upped its revenue forecasts for the year on the back of strong second quarter results from its new eczema and asthma injection Dupixent and its rare diseases and vaccines busines

News

NICE reviews number crunching methods as advanced technologi...

NICE is to review how it develops guidance on drugs, focusing on issues such as modifiers used in decision making and dealing with uncertainties in evidence.

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