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News
EMERGENCY APPEAL | TIF Rallies to Provide Earthquake Relief in Turkey and Syria
Among these people, the Thalassaemia International Federation (TIF) estimates that at least 2,000 patients with thalassaemia have been affected, 70% of whom are children and adolescents under the age of…
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TIF2023 | The Top International Conference On Thalassaemia Returns This November
The Conference, jointly organised by TIF, the Malaysian Haematology Society, and the Federation of Malaysia Thalassaemia Societies, will feature two parallel programmes – a Scientific Programme for Healthcare Professionals and…
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TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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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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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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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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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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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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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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