Rare diseases

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Alexion buys rare diseases biotech Syntimmune for up to $1.2...

Alexion is to buy rare diseases biotech Syntimmune and its experimental drug for a rare kind of anaemia, in a deal worth up $1.2 billion.

News

CSL Behring lung drug would cost NHS £8m per patient, says N...

NICE has rejected CSL Behring’s alpha1-proteinase inhibitor Respreeza for treating genetic emphysema in draft guidance, saying that it would cost a massive £8 million to give a patient a ye

News

Alexion prepares filings for new rare disease use for Soliri...

Alexion is preparing filings in major markets for its rare disease drug Soliris in a new indication after a positive readout in a rare neurological disease.

News

Spark/Novartis eye gene therapy nears EU market – but at wha...

Spark Therapeutics’ and Novartis Luxturna gene therapy for a rare inherited eye disease has been okayed by European regulators, paving the way for an EU licence in the coming months.

Views & Analysis

Raising awareness about rare diseases

A family tragedy has prompted Kay Parkinson, founder of the rare disease charity Alstrom Syndrome UK, to create a conference encouraging joined-up thinking to help improve treatments, care and serv

News

FDA rejects Pharming's Ruconest HAE label expansion

The FDA has rejected Pharming’s application to expand the licence of Ruconest, for a use as prophylaxis for patients with the serious rare disease hereditary angioedema (HAE).

Alexion buys rare diseases biotech Syntimmune for up to $1.2...

CSL Behring lung drug would cost NHS £8m per patient, says N...

Alexion prepares filings for new rare disease use for Soliri...

Spark/Novartis eye gene therapy nears EU market – but at wha...

Raising awareness about rare diseases

FDA rejects Pharming's Ruconest HAE label expansion

The long-delayed promise of the microbiome, with Sam Possemi...

How to meet your physicians where they are online

Rare diseases

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