Publications

The COVID-19 Pandemic & Haemoglobin Disorders (2020)

The COVID-19 Pandemic & Haemoglobin Disorders (2020)

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).
Thalassaemia from ‘A’ to ‘Z’: A comprehensive e-glossary for patients with thalassaemia (2019)

Thalassaemia from ‘A’ to ‘Z’: A comprehensive e-glossary for patients with thalassaemia (2019)

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…
Rapamycin

Rapamycin

  Update: 08 January 2021 No update available.   Update: 05 November 2020 No update available.   Update: 25 August 2020 No update available.   Update: 31 May 2020 No…
Mitapivat (AG-348)

Mitapivat (AG-348)

  Update: 08 January 2021  Interim data was presented at ASH2020: 12/13 evaluable patients have achieved a Hb increase of more than ≥ 1.0 g/dL after a median of 3.1…
Gene Therapy in β-thalassaemia and other haemoglobin disorders (2019)

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.
Apotransferrin

Apotransferrin

  Update: 08 January 2021 No update available.   Update: 05 November 2020 No update available.   Update: 25 August 2020 No update available.   Update: 31 May 2020 No…
Bone Marrow Transplantation in β-thalassaemia (2018)

Bone Marrow Transplantation in β-thalassaemia (2018)

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

Ferroportin inhibitors

VIT-2763   Update: 08 January 2021 No update available.   Update: 05 November 2020 No update available.   Update: 25 August 2020 Data presented at the EHA Congress in June…
SLN-124

SLN-124

Update: 08 January 2021 No update available.   Update: 05 November 2020 Initiated dosing in randomised, double-blind, placebo-controlled Phase 1 single ascending dose study of SLN124 in up to 24…
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