News
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SCIENCE NEWS | Research On Rare Genetic Disease Sheds Light On A Common Head And Neck Cancer
Patients with the rare genetic disorder Fanconi anemia lack certain elements of this repair system, making their cells incapable of removing lesions created by DNA-damaging chemicals, called aldehydes. And…
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PROFILE | After Major Advances In Hematology, Dr David Bodine Retires
When David M. Bodine, Ph.D., arrived at the US National Institutes of Health (NIH) for a postdoctoral fellowship in Arthur Nienhuis’s laboratory in the National Heart, Lung and Blood Institute (NHLBI),…
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CLINICAL NEWS | First Cohort of Patients Dosed In Phase 2 Study Of KP104 In PNH
KP104 is a first-in-class, bifunctional biologic designed to modulate complement activity through selective targeting of key intervention points in the alternative and terminal complement pathways. On July 28, 2022, the…
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DIAGNOSTICS | FDA Clears Baebies Disposable Enzyme Deficiency G6PD Test
Using a 50 µL blood sample (one drop of blood), FINDER delivers results approximately 15 minutes after sample introduction. Test results display G6PD enzyme activity in units per gram…
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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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