β-thalassaemia

POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells
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POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells

This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…
IRON BOY: SURVIVING β-THALASSAEMIA MAJOR by Arthur Bozikas
Patients’ stories

IRON BOY: SURVIVING β-THALASSAEMIA MAJOR by Arthur Bozikas

Where was my wife? I found myself sitting alone in front of the obstetrician, who my wife, Helen, and I had met for the first time only minutes earlier. But…
Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency
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Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency

The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
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FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy
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La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy

Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged…
Coming Up This Thursday: Discussion On Access To Innovative Therapies – The Reblozyl Case
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Coming Up This Thursday: Discussion On Access To Innovative Therapies – The Reblozyl Case

After receiving numerous questions from patients regarding the innovative treatment for β-thalassaemia Reblozyl (luspatercept), approved for use in the US, Europe, and Canada, TIF proceeds to organise an interactive, online…
EdiGene Announces First Patient Enrolment In Phase I Clinical Trial Of Gene-editing Therapy ET-01 For Patients With β-Thalassaemia
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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…
TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia
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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…
Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
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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…
It’s Here: The 4th Edition Of TIF Guidelines For The Management of TDT
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It’s Here: The 4th Edition Of TIF Guidelines For The Management of TDT

TIF, its Board of Directors, and its International Scientific Advisory Board proudly present the 4th Edition of the Federation’s most prestigious and renowned publication, the TIF Guidelines For The Management…
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