β-thalassaemia
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NOVITÀ
BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and…
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NOVITÀ
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…
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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…
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NOVITÀ
PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
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…
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NOVITÀ
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…
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NOVITÀ
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…
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NOVITÀ
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…
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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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NOVITÀ
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
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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