Patients

EdiGene Announces First Patient Enrolment In Phase I Clinical Trial Of Gene-editing Therapy ET-01 For Patients With β-Thalassaemia
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…
TIF Position Statement: Demanding Access To Existing And Upcoming Gene Therapies For β-Thalassaemia
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…
TIF Issues Charter of Priorities (CoP) For The Global PKD Patient Community
NOVITÀ

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
Clinical News

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
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 –…
CDC, FDA Authorize Third Dose Of COVID-19 Vaccines For The Immunocompromised In The US
Clinical News

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
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…
Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Clinical News

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
Clinical News

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
Clinical News

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…
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