Rare diseases | pharmaphorum

Friday 3 April 2026

Rare diseases

News

Could Spark Therapeutics’ merger with Roche finally get the go-ahead?

Could Spark Therapeutics’ merger with Roche finally get the ...

Spark Therapeutics’ merger with Roche has moved a step closer after the UK’s competition watchdog okayed the $4.8 billion deal, ahead of a regulatory deadline later today in the US.

News

French pharma company Sanofi

Sanofi axes diabetes R&D, focuses on 'transformative' therap...

Take a look at Sanofi’s balance sheet and it’s obvious which way the wind is blowing – sales in its once mighty diabetes franchise are sliding, while newer medicines such as its eczema and

Views & Analysis

Pricing-of-rare-disease-drugs-16x9-6

How does prevalence affect pricing of rare disease drugs?

New research from CRA looks at the relationship between rarity of a disease and the price countries are willing to pay for drugs to treat them.

Events

Partner Content

4th Annual Gene Therapy for Rare Disorders

Realize The Commercial Potential of Gene Therapies

News

Red blood cells moving in blood vessels with depth of field.

FDA approves Global Blood Therapeutics' sickle cell pill

Global Blood Therapeutics has seen its sickle cell disease drug approved by the FDA, promising to be the first treatment targeting the root cause of the condition.

Views & Analysis

FCS-A-life-limiting-condition

FCS: A life-limiting condition

GP Karishma Patel tells us about the daily challenges of living with the ultra-rare condition Familial Chylomicronaemia Syndrome (FCS), which prevents the body from breaking down fats.