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siltuximab

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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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FDA briefing document backs J&J’s one-dose COVID-19 vaccine

Company is seeking an emergency use authorisation in the US

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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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J&J submits one-dose COVID-19 vaccine to WHO for emergency use listing

In phase 3, J&J's vaccine was 66% effective overall at preventing moderate-to-severe COVID-19

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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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Verily and Janssen to collaborate on home-based COVID-19 immune response study

Study will collect information about how COVID-19 affects the immune system

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Janssen’s Erleada improves survival in certain advanced prostate cancer patients

Final analysis demonstrates a 35% reduction in the risk of death in mHSPC patients

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FDA schedules meeting on 26 February to review J&J’s one-dose COVID-19 vaccine

Vaccine candidate demonstrated an overall efficacy of 66% in phase 3

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J&J’s one-dose COVID-19 vaccine proves 66% effective in phase 3 trial

Vaccine was 57% effective against South African strain

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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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J&J completes enrolment for late-stage COVID-19 vaccine trial

Company announces that around 45,000 participants have been enrolled

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