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- PMLiVE

Rare Disease Day

Article: Engaging patient advocacy groups

The key to conducting effective rare disease research

Research Partnership

- 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

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

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

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

Statistics and Infographics for Rare Disease Day

Rare Disease Day 2019

Today is #RareDiseaseDay and is an opportunity to highlight some truly unbelievable/frightening statistics around rare diseases.

Cuttsy + Cuttsy

- PMLiVE

‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

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