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- PMLiVE

International Day

- PMLiVE

AstraZeneca’s Voydeya receives CHMP recommendation for rare blood disease PNH

Up to 20% of PNH patients treated with a C5 inhibitor experience clinically significant extravascular haemolysis

- PMLiVE

NHS to offer stem cell transplants to patients living with rare blood disorder

Over 600 adults in the UK are affected by transfusion-dependant thalassaemia

- PMLiVE

NHS to introduce world-first genetic blood-matching test for transfusion treatments

NHS Blood and Transplant will receive almost £1m in funding from NHS England

- PMLiVE

FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies

The company can now resume the enrolment and treatment of patients aged two to 17 years

- PMLiVE

Novartis’ iptacopan shows promise as rare blood disease treatment in phase 3 study

One to two people per million are diagnosed with paroxysmal nocturnal haemoglobinuria each year

- PMLiVE

bluebird bio’s beta-thalassaemia gene therapy receives FDA approval

Patients with the most severe form of the blood disorder develop life-threatening anaemia and have to receive regular blood transfusions

- PMLiVE

Celebrating the power of knowledge on International Thalassaemia Day 2022

This year’s ITD is on Sunday 8 May, promoting awareness about the global impact of the condition

- 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

Why blood donors and a solid national blood bank are vital

By Haifa Ungapen

- PMLiVE

Bluebird plays down report of EU gene therapy approval

Charity says Zynteglo (LentiGlobin) has already achieved a conditional marketing authorisation

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