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Archivi Autore: 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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Banca dati epidemiologica sulle malattie da emoglobina

Il progetto epidemiologico della TIF si concentra sulla raccolta di informazioni riguardanti le percentuali di soggetti portatori, le nascite premature e lo stato attuale delle strategie di controllo dell’emoglobinopatia nei sistemi sanitari dei paesi membri della TIF per β- e α-talassemia, HbE e anemia falciforme, le cui combinazioni costituiscono l’obiettivo ...

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Centri di riferimento per le emoglobinopatie

Si tratta di uno dei nuovi progetti della TIF intrapresi per il 2013. Il suo obiettivo è quello di sviluppare un database dei centri di riferimento per le emoglobinopatie in tutto il mondo, mappando i servizi che vengono forniti ai pazienti. Si tratta di un’estensione delle informazioni compilate nel progetto ...

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