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News
DRUGS | EMA Starts Pilot To Collect Information On Ongoing/Potential Shortages
To help address this issue, the European Medicines Agency (EMA) is starting a pilot to collect information on ongoing or potential shortages, according to the following criteria: The shortage…
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News
NEW APPROVAL | PYRUKYND® Gets EU Green Light For Pyruvate Kinase (PK) Deficiency
The European Commission has granted, on November 10, 2022, marketing authorization to Agios Pharmaceuticals for PYRUKYND® intended for the treatment of PK deficiency in adult patients. People with PK…
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Clinical News
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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News
NEW | Bluebirdbio Releases Resources For Patients On Gene Therapy Zynteglo®
To help people who are living with or caring for someone who has β-thalassaemia major get substantial information about Zynteglo®, bluebird bio, the company behind this ground-breaking therapy, has recently…
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Member News
THALASSAEMIA AND SICKLE CELL AUSTRALIA (TASCA) | Spring Newsletter 2022
Contents: Chair’s address Staff update TIF update TASCA Engagement In Memory of Lou Alborea TASCA kids’ page View the Newsletter HERE
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Clinical News
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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News
CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PK Deficiency 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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Clinical News
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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News
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News
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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