Innovative Therapies

IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
Clinical News

IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the…
European Commission Approves Reblozyl for the Treatment of Transfusion-dependent β-Thalassaemia
Clinical News

European Commission Approves Reblozyl for the Treatment of Transfusion-dependent β-Thalassaemia

Bristol Myers Squibb (BMS) & Acceleron Pharma announced today that the European Commission approved Reblozyl (luspatercept) for the treatment of Transfusion-Dependent β-#Thalassaemia (TDT) in adult patients. Reblozyl has the potential to…
Gene Therapy & Thalassaemia
Surveys

Gene Therapy & Thalassaemia

 Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells.   One of the novel…
eThalED Webinar Series: Access to New Therapies – Reblozyl
Webinars

eThalED Webinar Series: Access to New Therapies – Reblozyl

The 1st webinar for Medical Specialists organised in the context of TIF’s eThalED online course is here. Dr Antonio Piga, Professor of Paediatrics & Dean of the Medicine School at…
Explaining Gene Therapy in thalassaemia (2020)
Publications

Explaining Gene Therapy in thalassaemia (2020)

The second educational brochure from the TIF’s ‘Gene Therapy’ booklet series, providing an in-depth view of how this revolutionary therapy works in patients with β-thalassaemia major.
The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia
Webinars

The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia

9 patients with thalassaemia, members of TIF’s Patient Advocacy Group for Thalassaemia and Sickle Cell Disease (T-PAG), are discussing with the Thalassaemia International Federation the accessibility and affordability of the…
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
Clinical News

FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD

CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle…
Gene Therapy in β-thalassaemia and other haemoglobin disorders (2019)
Publications

Gene Therapy in β-thalassaemia and other haemoglobin disorders (2019)

An educational leaflet aiming to strengthen the knowledge and understanding of patients with thalassaemia and other haemoglobin disorders across the world on most aspects relevant to gene therapy.
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