Accueil » Archives de cet auteur: Catherine Skari

Archives de cet auteur: Catherine Skari

Gene Therapy & Thalassaemia Version 2.3: A TIF Survey

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 ...

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IGNORANCE ISN’T BLISS BY Jensen Chan

   »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 ...

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FDA Approves New Formulation for Ferriprox (Deferiprone) Twice-a-Day Tablets

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 ...

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TIF ITD2020 Webinar: The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia

    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. ...

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