Patients
EdiGene Announces First Patient Enrolment In Phase I Clinical Trial Of Gene-editing Therapy ET-01 For Patients With β-Thalassaemia
Νέα
2 εβδομάδες ago
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
Κλινικά Νέα
4 εβδομάδες ago
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…
TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community
Νέα
19 Αυγούστου, 2021
TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community
In the context of its educational and advocacy efforts within the field of other rare anaemias, beyond thalassaemia and sickle cell disease (SCD), and aiming, in particular, at the support…
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
Κλινικά Νέα
19 Αυγούστου, 2021
Mitapivat Granted Priority Review By The FDA For Pyruvate Kinase (PK) Deficiency
The U.S. Food and Drug Administration (FDA) has accepted Agios’ New Drug Application (NDA) for Mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. The NDA was granted…
TIF Presents The Federation’s Annual Report For 2020
Νέα
18 Αυγούστου, 2021
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 –…
CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
Κλινικά Νέα
17 Αυγούστου, 2021
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…
TIF e-Academy: Reimagining Thalassaemia and Sickle Cell Disease Education
Νέα ΔΟΘ
30 Ιουλίου, 2021
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…
Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Κλινικά Νέα
30 Ιουλίου, 2021
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…
Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
Κλινικά Νέα
12 Ιουλίου, 2021
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…
Clinical Trials Update: The June 2021 Edition
Κλινικά Νέα
5 Ιουλίου, 2021
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…