Patients
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Clinical News
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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NOVITÀ
EdiGene Announces First Patient Enrolment In Phase I Clinical Trial Of Gene-editing Therapy ET-01 For Patients With β-Thalassaemia
The Chinese biotechnology company EdiGene announced yesterday that the first patient was enrolled in the multicentre, Phase I clinical study of its investigational gene editing hematopoietic stem cell therapy ET-01…
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Clinical News
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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NOVITÀ
PYRUVATE KINASE DEFICIENCY | TIF Issues Charter of Priorities For The Global PKD Patient Community
This newly launched document aspires to bring together available information about PKD in an effort to identify gaps, and to make useful imperative suggestions so as to understand what changes…
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Clinical News
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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NOVITÀ
TIF Presents The Federation’s Annual Report For 2020
“Our commitment to unite the patient voice and to advocate at the highest possible levels for equal access to all treatment options remains our number one priority.“ TIF President –…
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NOVITÀ
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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TIF News (IT)
TIF e-Academy: Reimagining Thalassaemia and Sickle Cell Disease Education
Read. Learn. Empower. The Thalassaemia International Federation´s internationally acclaimed Educational Programme is a fundamental component of TIF´s work and is specifically designed for and addressed to the ever-growing learning needs…
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NOVITÀ
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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NOVITÀ
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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