ultra-rare diseases

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GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter ...

GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE.

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London, UK - January 8, 2015: City of London panorama includes high street with office and governments buildings

Orchard’s gene therapy gains UK ‘promising innovative medici...

A UK biotech start-up has gained a sought after ‘promising innovative medicine’ (PIM) designation for its ultra-rare disease gene therapy.

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Alexion price cut opens up Strensiq to more patients

Managed access scheme will control costs to NHS.

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Outrage over Duchenne drug price reinflamed by PTC's $35,000...

Families affected by the rare muscle wasting disease Duchenne muscular dystrophy will still have to pay a huge premium for Emflaza, a newly-approved drug based on an old steroid that helps

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Sucampo buys rare disease firm Vtesse

Sucampo gains rights to a drug for rare genetic disorder.

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Alexion appoints former Baxalta chief Hantson as CEO

Former AZ chief David Brennan expected to take over as chairman in May.

GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter ...

Orchard’s gene therapy gains UK ‘promising innovative medici...

Alexion price cut opens up Strensiq to more patients

Outrage over Duchenne drug price reinflamed by PTC's $35,000...

Sucampo buys rare disease firm Vtesse

Alexion appoints former Baxalta chief Hantson as CEO

LSX2026: Addressing adult eye cancer, with Roi Raz

Using AI-driven synthetic personas to take your insights fur...

ultra-rare diseases

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