Rare Disease

News

Scotland first in Europe to back MSD drug for VHL tumours

Scotland has become the first country in Europe to recommend coverage of MSD’s Welireg as a treatment for some people with von Hippel-Lindau disease (VHL), a rare genetic

R&D

Navigating EAPs: Clinical trials and the path to investigati...

Medical breakthroughs have always been milestones lighting our path to better health.

Patients

Partner Content

The Multilayered Impact of Rare Disease on Relationships

Pharma marketers: rare conditions can impact all types of relationships and one's social life.

News

Travere weaker after Filspari fails confirmatory trial

Shares in Travere Therapeutics have lost more than 40% of their value after the company said that a trial designed to confirm the benefits of its recently approved Filspar

News

AZ's Alexion inks wide-ranging AI agreement with Verge

AstraZeneca has made a sizeable play in applying artificial intelligence to drug discovery in its rare disease unit Alexion, partnering with Verge Genomics in a deal that

News

FDA starts review of Roche's PNH drug crovalimab

Roche could be just months away from FDA approval of its anti-complement C5 antibody crovalimab, after the US regulator started a review of its marketing application as a

Scotland first in Europe to back MSD drug for VHL tumours

Navigating EAPs: Clinical trials and the path to investigati...

The Multilayered Impact of Rare Disease on Relationships

Travere weaker after Filspari fails confirmatory trial

AZ's Alexion inks wide-ranging AI agreement with Verge

FDA starts review of Roche's PNH drug crovalimab

Prevention for a healthier, more productive society

Health Trends 2025: Reimagining What's Possible

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