Rare Disease

News

Sanofi extends its Pompe range with first Nexviazyme OK

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder.

News

Sanofi swoops on partner Translate, buying the mRNA firm for...

Sanofi has been forging ever-closer ties with its mRNA partner Translate Bio since the two started working together in 2018, and has now gone all in, offering $3.2 billion to buy the compan

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Ipsen forges $1bn alliance with Exicure on neurodegenerative...

Ipsen's new chief executive David Loew pledged to ramp up spending on the company's pipeline when he took the helm last year, and a flurry of licensing deals in the last few weeks shows tha

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Boost for AZ as Ultomiris gets CHMP nod for expanded label

Fresh from its takeover of Alexion, AstraZeneca has picked up a recommendation in the EU for an expansion of the label of Ultomiris, one of the main assets behind the

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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

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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