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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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ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
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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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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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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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A HISTORIC BREAKTHROUGH | UNICEF Signs First-Ever Malaria Vaccine Supply Contract With GSK
The contract will enable UNICEF to deliver more than 18 million doses of the RTS,S vaccine – also known under the commercial name Mosquirix – in order to reduce the…
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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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NEW PUBLICATION | Short Guide For the Management of TDT (2nd Edition)
The 4th edition of the original full-length Guidelines, published in June 2022, includes a full review of the evidence supporting almost each recommendation for physicians to know all the facts…
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