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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

UK budget includes £1bn biopharma investment package

HHS doubles down on controversial peptides plan

Lilly's triple G overpowers Ozempic in diabetes trial

UK puts £100m into "world-first" AI biosecurity facility

US gives $99m for Ebola jab project, as cases spread in Africa

Oncology

On the benefits of earlier oncological intervention, with Johnson & Johnson

On the benefits of earlier oncological intervention, with Jo...

As ESMO 2026 came to a close, pharmaphorum web editor Nicole Raleigh spoke with Johnson & Johnson Innovative Medicine’s Carolyn Sousa and Henar Hevia

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Digital

Discover a new era of healthcare panel engagement

Sponsored

Discover a new era of healthcare panel engagement: Grounded ...

A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.

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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