Publications

A  Haemoglobinopathy Patient Care Pathway suggesting changes to be made during the outbreak of COVID-19 in the admittance of patients to haemoglobinopathy day care centres /units /clinics.

An informational leaflet about the new COVID-19 with a special focus to the challenges it presents for patients affected by thalassaemia and sickle cell disease (SCD).

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 “Thalassaemia from A to Z” electronic glossary is a comprehensive, easy to use, educational tool for thalassaemia, including definitions of all important concepts on the prevention, management, treatment, organ complications, and plenty of other aspects related to the disease.

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 Rapamycin, a drug widely used for almost 20 years to protect organ transplant patients, has been found to reduce, in mice, the build up of toxic proteins that destroy ...

  Update: 31 January 2020 The clinical proof-of-concept has been established based on a preliminary analysis of the Phase 2 trial of Mitapivat (AG-348) in patients with non-transfusion-dependent thalassaemia announced at the 61st Annual Meeting of the American Society of Haematology. The Phase 2 study has enrolled 12 of the ...

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.

ARU-1801 Gene Therapy (SCD)   Update: 31 January 2020 The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, an experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassaemia. This designation means eligibility for a  priority review voucher once the FDA approves a biologics ...

  Update: 31 January 2020 No update available   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019  Apotransferrinhas a physiological role in the transportation and distribution of iron among the ...

An educational leaflet with important information for β-thalassaemia patients on hematopoietic stem cell transplantation (HSCT), more commonly known as bone marrow transplantation (BMT).