orphan disease

Views & Analysis

Who will be the biggest pharma players in orphan drugs?

New analysis from GlobalData has identified the predicted top 15 pharma players in 2025 for organ drugs, with Roche coming out in the lead.

Views & Analysis

Balancing expectations: An unmet need in the orphan disease ...

There are between 6000 and 8000 rare diseases in the world, and one in ten people in Europe and the US have a rare disease; 75% of rare diseases affect children, and 30% of rare disease patients di

Partner Content

Hear from HealthTech Healx which raised $10m to accelerate d...

SMi Reports: AI HealthTech Company Healx, who raised $10m to advance drug dis

News

FDA approves Teva's Huntington's Disease drug

First treatment approved for Huntington's Disease in almost a decade.

Views & Analysis

Funding rare disease treatments in the UK – navigating the l...

With the appraisal process for highly specialised treatments in the UK described by one NHS insider as 'impenetrable', and The MPS Society's (The Society for Mucopolysaccharide Diseases) recent

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