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- PMLiVE

Galafold

- PMLiVE

Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Company to press ahead with phase 3 trial

- PMLiVE

Alexion’s Soliris adds new approval in rare disease NMOSD

First ever drug approved for rare autoimmune disease

- PMLiVE

Acer Therapeutics hit by FDA filing rejection

Shares plunge as regulator calls for new trial

- 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

Spinraza managed access deal in England breaks deadlock for Biogen

Patient groups remain wary, call for changes to NICE

- 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

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

- PMLiVE

Biogen’s Spinraza overpriced, says ICER

Cost watchdog warns high price puts pressure on US health system

- 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

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