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Rare diseases

News

Amicus

Amicus set for share of Fabry market after NICE approval

England and Wales’ cost effectiveness watchdog NICE has given a final recommendation to a new treatment for the rare Fabry disease.

News

Alexion

Alexion drug fails in kidney transplant trial

Soliris fails when used to treat post-transplant complications.

News

European Medicines Agency London

Sarepta files controversial Duchenne drug in EU

US biotech seeks conditional approval for eteplirsen in EU.

News

edinburgh

Doubts over future of rare drug fund in Scotland

Funding could be unsustainable, says review.

News

actelion

Sanofi outbids J&J for Actelion merger - reports

Reports suggest Sanofi could pay $30 billion for Swiss biotech.

News

group of chemical scientists working at the laboratory.

Shire to build US rare disease R&D hub

New facility in Cambridge, Massachusetts.

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Editor's Picks

Vertex buys Crinetics for $10bn in endocrinology drive

UK first in Europe to clear Boehringer's IPF drug Jascayd

NICE aims to cut funding for Amgen lung cancer drug Lumykras

Island Pharma drug cleared for Bundibugyo Ebola use

NICE backs NHS use of non-invasive endometriosis tests

News

Dr Anker Lundemose

UK's Mission divests kidney drug to Dimerix for $292m

Mission Therapeutics has struck a deal to out-license a drug for acute kidney injury to Australia's Dimerix, sharpening its focus on CNS diseases.

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Digital

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company