Rare Disease

News

NHS sets aside £340m for new Innovative Medicines Fund

NHS England has matched its financial backing of the Cancer Drugs Fund (CDF) with an equivalent pot of money for a new Innovative Medicines Fund (IMF) that will provide early access to cutt

News

Bluebird claims EU nod for rare disease therapy Skysona

Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the

News

Final hurdle falls as UK backs AZ's $39bn takeover of Alexio...

The UK's competition regulator has approved AstraZeneca's $39 billion acquisition of US-based drugmaker Alexion, without opting for a broader inquiry into the deal.

News

Novo Nordisk bulks up in rare diseases with $1.2bn Prothena ...

Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis.

News

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

NICE rejects Orchard's gene therapy for rare childhood disea...

Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.

News

MHRA grants bluebird 'innovation passport' for sickle cell t...

bluebird bio's sickle cell disease (CD) gene therapy LentiGlobin is the latest recipient of an 'innovation passport' introduced in the UK earlier this year to speed up NHS access to promisi