Patients
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Actualités
NEW HOPE FOR THALASSAEMIA/SCD | UK Trial Of Lab Grown Red Blood Cells Begins
The Restore trial, led jointly by NHS Blood and Transplant and the University of Bristol, is studying the lifespan of the lab grown cells compared with infusions of standard red blood…
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Actualités
CDC | Many Children With Sickle Cell Anaemia In The US Not Receiving Lifesaving Screening And Treatment
According to a recent CDC study, fewer than half children with the disease in the US get the necessary screening, and only about half or fewer get treatment with hydroxyurea…
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CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PKD in Adults
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy…
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SAFER BLOOD | EU Patients Have Begun Receiving Transfusions With Hemanext ONE® RBC Processing & Storage System
Doctors have begun transfusing patients suffering from hematological malignancies using RBCs processed and stored with the Hemanext ONE® system, as part of a post-market clinical study in Bergen, Norway. This is…
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Actualités
SICKLE CELL DISEASE | African Health Ministers Launch Drive to Curb SCD’s Toll
This effort, backed by the African Regional Office of the World Health Organization (WHO), aims to curb SCD΄s toll in a region that has historically been hit hard by this…
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Actualités
THALASSAEMIA CHAMPIONS PODCAST | The 3rd And Final Episode Is Now Available
With open dialogue-formatted episodes, featuring thalassaemia patient advocates, mental health professionals, and other special guests, this show explores everything from psychosocial anxiety and relationship issues to treatment-related fears, while striving…
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BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and…
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Actualités
Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease
Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of…
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Actualités
POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells
This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…
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