News
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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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UNITED Onlus | The Italian Federation Of Thalassaemia Welcomes Newly Elected BoD
The assembly was held in the conference room of the Zone Hotel in Rome. The members of the new board are: Vindigni Raffaele, Outgoing President, elected Vice President Vicario Martinelli…
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OUT NOW | 2021 TIF Annual Report Has Been Published
The 2021 Annual Report will cover the following and more: Message by TIF’s President and Executive Director 2021 in Numbers Country Activities TIF International Conference Global Thalassaemia Review 2021 Strategic…
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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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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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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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