Rare diseases | pharmaphorum

Skip to main content

Monday 6 April 2026

BlueSky linkedin youtube

header links

Get Newsletters
Media Kit

Pharmaphorum menu first category

News
Views & Analysis
Deep Dive
Webinars
Podcasts
Videos
Spotlight on
Events

close

SECTION

News
Views & Analysis
Deep Dive
Webinars
Podcasts
Videos
White Papers
Events

Channels

Channels

Sales & Marketing
R&D
Digital
Market Access
Oncology
Patients
Spotlight On

Channels

Sales & Marketing
R&D
Digital
Market Access
Oncology
Patients
Spotlight On

SUB NAVIGATION

Live Coverage
Partner Content
About us
Work for us
Contribute
Contact us
Advertise

Newsletters and Deep Dive digital magazine

youtube linkedin BlueSky

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.

Load more results

Editor's Picks

Novartis snaps up PI3Kα breast cancer drug in $3bn deal

US court says pharma giants must face False Claims suit

Meningitis outbreak in UK prompts call to widen vaccination

US judge rules Kennedy's vaccine changes 'unlawful'

Generic semaglutide in India, and other weight-loss news

Sales & Marketing

Interview with Craig Ackerman Reuters at Pharma USA 2026

Biotech is back, with Craig Ackerman

In Philadelphia this month, pharmaphorum spoke with Craig Ackerman, a partner at the Alexander Group, about launches, AI best practices, and more.

/themes/custom/pharmaphorum_theme/library/img/article-label-image/ondemand.png

Sales & Marketing

Using AI-driven synthetic personas to take your insights further

Sponsored

Using AI-driven synthetic personas to take your insights fur...

As AI continues to reshape the healthcare landscape, pharma teams are beginning to leverage synthetic personas.

Pharmaphorum Menu

Digital
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
Market Access
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
Oncology
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
Patients
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
R&D
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
Sales & Marketing
- News
- Views & Analysis
- Deep Dive
- Webinars
- Podcasts
- Video
- White Papers
- Event
Spotlight on

Sign up for email newsletters and Deep Dive

Get Newsletters

youtube linkedin BlueSky

About us Advertising Contact us

Contribute Cookie Policy Privacy Policy

Terms & Conditions Work for us

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

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