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- PMLiVE

fast track appraisal

- PMLiVE

Novartis and NHS England reach ‘landmark’ deal for Zolgensma gene therapy for SMA

Children born with SMA type 1 typically have a life expectancy of around two years

- PMLiVE

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?

- 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

UK hits ‘significant milestone’ as 15 million people receive their first COVID-19 vaccine dose

The NHS also announced that five additional large-scale vaccination centres will open this week

- PMLiVE

NICE turns down bluebird bio’s gene therapy Zynteglo

Draft guidance does not recommend routine NHS funding for beta thalassaemia treatment

- PMLiVE

Gilead’s Jyseleca is given a NICE recommendation for rheumatoid arthritis

JAK inhibitor approved for use in moderate-to-severe RA

- 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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