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- PMLiVE

Rare Disease Day 2021

- PMLiVE

NHS England wants money-back guarantee on Orkambi

Vertex resisting real-world data proposal

- PMLiVE

Boehringer eyes rare disease use for respiratory blockbuster

Would help support respiratory franchise

- PMLiVE

Speedy approval for Pfizer’s Onpattro rival

Approved in two different formulations

Biomarin

EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

- PMLiVE

Bluebird’s myeloma CAR-T safety ‘could support outpatient use’

Expects to file with the FDA next year

- PMLiVE

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

Bluebird plays down report of EU gene therapy approval

Charity says Zynteglo (LentiGlobin) has already achieved a conditional marketing authorisation

- PMLiVE

Unruffled by ‘outlier’ claims, Vertex CEO upbeat on Orkambi breakthrough

NHS England going public with price makes deal impossible, says Leiden

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

- PMLiVE

Afinitor and Uptravi gain funding from NHS England

Rare disease treatments navigate tricky market access route

- PMLiVE

Chugai gains FDA breakthrough status for rare CNS treatment

Adds to company's BTD roll-call

- PMLiVE

Roche gains EU PRIME status for Spinraza rival

Oral treatment joins Novartis' gene therapy challenger

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