Clinical News
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CLINICAL ALERT: New study highlights ‘unsafe’ T2* MRI method being used for iron overload assessment in thousands of patients
New study highlights ‘unsafe’ T2* MRI method being used for iron overload assessment in thousands of patients. TIF became aware of the results from a new study, to be presented…
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UK & Wales: NICE Recommends First Treatment In Two Decades For Sickle Cell Disease
For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK National Health Service (NHS). Crizanlizumab (Adakveo) by Novartis…
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BLOOD TRANSFUSION | Hemanext Begins Rollout of Hemanext ONE® RBC Processing And Storage System In Europe
Physicians in Norway have initiated recruitment of chronically transfused patients with haematological malignancies, including myelodysplastic syndrome (MDS), to receive transfusions using RBCs processed and stored with the Hemanext ONE® system.…
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TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia
The Thalassaemia International Federation (TIF) is expressing its grave disappointment over the recent announcement of Bluebird Bio regarding the “wind-down” of its operations in Europe and the exclusive disposal of…
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MITAPIVAT | FDA Grants Priority Review To New Drug For Pyruvate Kinase Deficiency
The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from…
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CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
The U.S. Food and Drug Administration (FDA) and the U.S. Centers for Disease Control and Prevention (CDC) have both recommended on August 16 that certain immunocompromised people receive a third…
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Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (Voxelotor) if the U.S. Food and Drug Administration (FDA) approves a supplemental new drug application (sNDA) submitted by…
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Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
The European Medicines Agency (EMA)’s safety committee (PRAC) has concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukaemia (AML). Zynteglo, a gene therapy…
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Clinical Trials Update: The June 2021 Edition
TIF provides you with comprehensive and up-to-date information on developing drugs and therapies for thalassaemia and sickle cell disease (SCD) currently in clinical trials. Our most recent June 2021 update…
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