Gene Therapy

Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
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Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?

In the aftermath of the withdrawal of Zynteglo, the first gene therapy for patients with β-thalassaemia, from Europe, TIF´s Executive Director, Dr Androulla Eleftheriou, reflects on the viability of the…
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…
Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
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Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies

bluebird bio, Inc. today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin…
Ali Sattarpour:  »Thalassaemia did not stop me from living a normal life »
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Ali Sattarpour:  »Thalassaemia did not stop me from living a normal life »

In occasion of the International Thalassaemia Day 2021, Ali Sattarpour, 45, France, Fédération SOS GLOBI Vice President and TIF’s Thalassaemia Patient Advocacy Group (T-PAG) Member, recounts a life of struggles,…
TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia
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TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia

The Thalassaemia International Federation (TIF) expresses its disappointment about the outcome of the reimbursement negotiation process concerning gene therapy (Zynteglo™) for transfusion-dependent thalassaemia patients, announced last week. The Federation’s distress…
A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic
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A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic

The expansion of management options and therapies for patients with thalassaemia is nowadays a reality, thanks to the application of new, revolutionary scientific procedures. However, patients’ understanding on the distinct…
TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®
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TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®

On February 12, in an appraisal consultation document, the UK National Institute for Health and Care Excellence (NICE) recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) gene therapy for the…
GoCART Coalition: EHA and EBMT Join Forces To Position Europe In The Lead Of The Global Cellular Therapy Field
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GoCART Coalition: EHA and EBMT Join Forces To Position Europe In The Lead Of The Global Cellular Therapy Field

The European Hematology Association (EHA) and the European Society of Blood and Marrow Transplantation (EBMT) announce a strategic partnership to empower Europe to become a global leader in the cellular…
Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges
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Advances in the Care & Cure of Thalassaemia: New Hopes & Challenges

TIF, in collaboration with Thalassemics India and Thalassemia Patients Advovacy Group – India, conducted on June 12, 2020 a Webinar on the novel treatments emerging for thalassaemia and the hopes…
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