Publications

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: 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 red blood cells in β-thalassaemia. Investigators are moving ...

  Update: 25 November 2019 No update available.   Update: 27 September 2019 DRIVE PK is an ongoing global, open-label, Phase 2, safety and efficacy study evaluating Mitapivat in adults with PK deficiency who do not receive regular transfusions. It is the first clinical trial in adults with PK deficiency. ...

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.

CRISPR gene editing (SCD)   Update: 25 November 2019 CRISPR Therapeutics and Vertex Pharmaceuticals announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001. 1 patient with severe sickle cell disease (SCD) received CTX001 in mid-2019 and data for this ...

  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 body organs. Apotrasferrin has received orphan drug designation ...

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

VIT-2763   Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 Results announced during 24th Annual Congress of the European Hematology Association show that treatment with single and multiple oral doses of VIT-2763 were well tolerated in healthy subjects, ...

  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 No update available.   Update: 29 March 2019 SLN124 has been submitted to the UK Medicines and Healthcare Products Regulatory Agency ...

  Update: 25 November 2019 No update available.   Update: 27 September 2019 No update available   Update: 30 July 2019 Sandoz, a Novartis division focusing on biosimilars have enrolled the first patient in an intergrated Phase I/III study for the development of denosumab for use in osteoporosis. Sources: https://www.novartis.com/news/media-releases/sandoz-announces-first-patient-enrolled-clinical-study-proposed-biosimilar-denosumab-osteoporosis ...