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Tag Archives: Patients

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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FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD

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 ...

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Reblozyl (luspatercept-aamt) Receives Positive CHMP Opinion for the Treatment of Adults with β-Thalassaemia

  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. ...

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COVID-19: TIF Releases Position Statement on the Inclusion of Thalassaemia & SCD Patients in the High-Risk Population

  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 ...

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