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 forward to design a small proof-of-concept clinical trial ...

  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. Mitapivat is an investigational, first-in-class, oral, small molecule ...

This is how this year’s voting results were shaped: Universal access to quality thalassaemia healthcare services: Building bridges with and for patients (Winning theme, 196 votes) Patients’ voice at the frontline: Advocating for a better future in thalassaemia care and management (112 votes) The globalization of thalassaemia – Common challenges ...

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CRISPR gene editing (SCD)   Update: 27 September 2019 Enrollment in Phase 1/2 study of CTX001 in patients with severe SCD is ongoing. Based on the progression of the program, CRISPR Therapeutics expects to obtain preliminary safety and efficacy data in late 2019. The first patient has been treated in ...

  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 from EMA A proof of concept study in ...

  This is how this year’s voting results were shaped: Universal access to quality thalassaemia healthcare services: Building bridges with and for patients (Winning theme, 196 votes) Patients’ voice at the frontline: Advocating for a better future in thalassaemia care and management (112 votes) The globalization of thalassaemia – Common ...

يركز مشروع علم الأوبئة التابع للاتحاد الدولي للثلاسيميا على جمع المعلومات حول معدلات حاملى المرض والولادات المتوقعة والوضع الحالي المعني باستراتيجيات التحكم بأمراض امراض الهيموجلوبينبداخل الأنظمة الصحية الخاصة بالدول الأعضاء المختلفة لدى الاتحاد الدولي للثلاسيميا لمرضى الثلاسيميا بيتا والثلاسيميا ألفا، والأنيميا المنجلية ، و هي تشكل مجموعات التركيز الرئيسي لهذا ...

هذا المركز هو احد مشاريع الاتحاد الدولي للثلاسيميا الجديدة التي تم تنفيذها لعام 2013 وهدفه هو تطوير قاعدة بيانات لمراكز مرجعية لامراض الهيموجلوبين في جميع أنحاء العالم ، ورسم الخرائط للخدمات التي تقدم للمرضى. وهو امتداد للمعلومات التي جمعها مشروع ENERCA-3 ومناقشات EUCERD ، والتي تركز على تعزيز المراكز المرجعية ...