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Article: Engaging patient advocacy groups

The key to conducting effective rare disease research

Research Partnership

- PMLiVE

Labour party plans to abolish NHS prescription charges

Comes amid increased focus on drug pricing in the US

- PMLiVE

Roche’s Hemlibra scores broader NHS funding

Expands to around 2,000 patients in England

- 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

Orkambi row: government now considering Crown Use licensing

MPs call for radical solutions as deadlock remains

- PMLiVE

Roche Diagnostics among beneficiaries as NHS fast-tracks more tech

AHSNs will be responsible for regional uptake

- PMLiVE

NHS England wants money-back guarantee on Orkambi

Vertex resisting real-world data proposal

- PMLiVE

Still no breakthrough on Orkambi talks, MPs want answers

As other price deals go through, Orkambi stalled again

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