Tag Archives: Featured

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 therapies  for thalassaemia that has stood out recently has undoubtedly been the first and only, one-time, groundbreaking gene therapy for transfusion ...

On 14 June 2020 WHO and all countries worldwide will celebrate World Blood Donor Day!   The campaign theme for this year’s World Blood Donor Day is “Safe blood saves lives” with the slogan “Give blood and make the world a healthier place”. The idea is to focus on the ...

“I was shocked at the huge discrepancy between how common thalassaemia is and how few people actually know about it.“ Jensen Chan, a University student from Singapore, explains how his brother, a thalassaemia carrier, inspired him to learn more about this blood disorder and start a campaign to increase public ...

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., today announced that the U.S. Food and Drug Administration (FDA) has approved Ferriprox® (deferiprone) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, when current chelation therapy is inadequate. The new formulation of ...

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 Cell Disease (SCD). As with the FDA’s breakthrough ...

As the online celebrations for the International Thalassaemia Day 2020 are at their peak, TIF decided to publish today as a gift to its worldwide patient communities a new guide entitled “COVID-19 & Haemoglobin Disorders: A Classification of Risk Groups & Other Considerations“. Patients with thalassaemia and sickle cell disease ...

  On 30 April 2020, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product REBLOZYL, intended for the treatment of adults with beta-thalassaemia or transfusion-dependent anaemia associated with myelodysplastic syndromes (MDS). The applicant for this medicinal product is Celgene Europe BV. ...

The 1st webinar for Medical Specialists organised in the context of TIF’s eThalEd online course has just arrived! Dr Antonio Piga, Professor of Paediatrics & Dean of the Medicine School at San Luigi University Hospital of the Turin University, will be the host of this introductory webinar entitled ”Access to New Therapies: Reblozyl”. ...

TIF expresses major concerns over the announcement of the United States to suspend financial support for the World Health Organisation (WHO) until completion of a review of the organisation’s activities related to the COVID-19 pandemic.

The first UK vaccine candidate to prevent coronavirus infection is set to begin clinical testing in human volunteers  today. Its developers at the University of Oxford hope to have 1m vaccine doses ready by September to carry out large-scale clinical trials during the autumn and, if all goes well, to ...