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- PMLiVE

Wakix

- PMLiVE

The five-step, rare disease-ready checklist

How can a commercial team find success in the rare disease area?

- PMLiVE

bluebird bio marks Rare Disease Day 2021 with launch of patient testimonial video

Personal stories from nine patients from seven European countries, aged between 4 and 61, shed light on the impact of living with thalassaemia

- PMLiVE

Protalix, Chiesi’s monthly treatment PRX-102 shows benefit in Fabry disease

Fabry disease is a rare disorder in which patients inherit a deficiency of the α–Galactosidase–A enzyme

- PMLiVE

UK introduces Rare Diseases Framework to improve the lives of people with rare diseases

bluebird bio releases a video to raise awareness – Rare Disease Day 2021 is on 28 February

- PMLiVE

Why we need to look at rare diseases in a different way

28 February 2021, Rare Disease Day aims to raise awareness of rare diseases and the impact they have on patients’ lives.

Origins – The Patient Focused Specialists

- PMLiVE

Rare Disease Day – why it’s important to show your stripes

Nucleus Global's EU Strategy Director, Amanda Henkel, writes about the importance of continuing to raise awareness of rare diseases

Nucleus Global

- PMLiVE

EMA begins rolling review of CureVac’s COVID-19 vaccine

Investigational vaccine is currently being evaluated in a phase 2/3b clinical trial

regeneron headquarters

EMA launches rolling review of Regeneron’s COVID-19 antibody cocktail

Study suggests REGN-COV2 can reduce viral load in non-hospitalised patients with COVID-19

- PMLiVE

CHMP recommends EU approval for Amarin’s cardiovascular risk reduction drug

The drug is recommended for use in adult patients who are already receiving statin treatment

- PMLiVE

New dosing option for AZ’s Imfinzi approved in the EU and UK

Additional option extends dosing from two weeks to four weeks

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AZ seeks EU approval for its Oxford University-partnered COVID-19 vaccine

CHMP could reach a decision on opinion by 29 January

- PMLiVE

bluebird bio to separate oncology and rare diseases units

New oncology company will be led by current bluebird bio CEO Nick Leschly

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