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Author Archives: tifwp

      IS BETA – THALASSAEMIA OR SICKLE CELL DISEASE IN YOUR PIPELINE?   LEARN ABOUT PATIENTS’ EXPECTATIONS, PERSPECTIVE, CHALLENGES AND BURDEN OF DISEASE BY ENGAGING WITH TIF’s INTERNATIONAL PATIENT ADVOCACY GROUP!   The Thalassaemia International Federation (TIF) is delighted to announce the establishment of TIF’s Patient Advocacy Group ...

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Rapamycin

  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 ...

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Mitapivat (AG-348)

  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 ...

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Sickle Cell Disease Update

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 ...

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Apotransferrin

  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 ...

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International Thalassaemia Day 2019 – Get inspired, get involved!

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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International Thalassaemia Day 2019 – Get inspired, get involved!

  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 ...

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Ferroportin inhibitors

VIT-2763   Update: 27 September 2019 No update available   Update: 30 July 2019 Results announced during 24th Annual Congress of the European Hematology Association show that treatment with single and multiple oral doses of VIT-2763 were well tolerated in healthy subjects, decreasing the iron levels in circulation. A proof ...

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SLN-124

  Update: 27 September 2019 No update available   Update: 30 July 2019 No update available.   Update: 30 May 2019 No update available.   Update: 29 March 2019 SLN124 has been submitted to the UK Medicines and Healthcare Products Regulatory Agency (MHRA). Subject to approval from the MHRA, Silence ...

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