Tag archives : Patients

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 ...

   »Growing up as an avid member of the The Boys’ Brigade, my younger brother, Neilsen, was physically active and never had any fitness or health issues. However, at age 16, he began to develop symptoms of anaemia, such as easy fatigue and shortness of breath during exercise. Our concerned ...

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 ...

    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 new and innovative Therapies for the disease, as well as the COVID-19 pandemic challenges in their respective countries. ...

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 ...

A TIF-compiled document aiming to provide a division of risk levels for thalassaemia and SCD patients with regards to the ongoing COVID-19 pandemic.

  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 SARS-CoV-2 infection presents particular challenges and great risks to patients with thalassaemia and sickle cell disease (SCD) and, if their appropriate clinical management as described in International Guidelines is not safeguarded in the context of national policies developed for fighting COVID-19, their health and quality of life will ...