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Author Archives: tifwp

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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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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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 ...

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Epidemiologische Datenbank zu Hämoglobinkrankheiten

Das epidemiologische Projekt des TIF konzentriert sich auf die Erhebung von Daten über Krankheitsträger, erwartete Geburten und den aktuellen Stand der Strategien zur Bekämpfung der Hämoglobinopathie in den Gesundheitssystemen der verschiedenen Ländern, die Mitglied der TIF sind. β- und α-Thalassämie, HbE und Sichelzellkrankheit (SCD) sowie Kombinationen bilden den Schwerpunkt dieser ...

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Referenzzentren für Hämoglobinopathie

Dies ist eines der neuen Projekte der TIF für das Jahr 2013, dessen Ziel es ist, eine Datenbank für Referenzzentren für Hämoglobinopathie auf der ganzen Welt einzurichten und eine Übersicht der Dienstleistungen zu erstellen, die sie für Patienten erbringen. Dies ist eine Erweiterung der Informationen des ENERCA-3-Projekts und der Beratungen ...

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