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spinal muscular atrophy

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Rare Disease Day in a COVID-19 environment

For many people and families living with a rare disease, their journey can be very complex. At times, that journey can even be scary, frustrating and confusing. However, when your...

Lucid Group Communications Limited

- PMLiVE

The five-step, rare disease-ready checklist

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

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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

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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

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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

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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

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Novartis partners with Bill & Melinda Gates Foundation on ‘accessible’ sickle cell gene therapy

Project will aim to discover and develop a gene therapy for sickle cell disease

- PMLiVE

Rare genetic diseases – what’s in store for 2021?

Why personalised medicine based on our own genomes is the future of healthcare

- PMLiVE

Novartis, Bayer announce separate agreements to bolster COVID-19 vaccine manufacturing

Novartis will help manufacture Pfizer/BioNTech's vaccine and Bayer will do the same for CureVac's vaccine

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Novartis hit by ‘COVID-19-related lockdowns’ in Q4

Sales grew in the quarter but profits fell short of forecasts

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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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