Home » Author Archives: tifwp

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 ...

Read More »

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 ...

Read More »

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 ...

Read More »

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 ...

Read More »

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 ...

Read More »

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 ...

Read More »

Τράπεζα δεδομένων για τις διαταραχές της αιμοσφαιρίνης

Το επιδημιολογικό έργο της ΔΟΘ εστιάζει στη συλλογή πληροφοριών σχετικά με τα ποσοστά φορέων, τις προβλεπόμενες γεννήσεις και την τρέχουσα κατάσταση των στρατηγικών για τον έλεγχο των αιμοσφαιρινοπαθειών στα συστήματα υγείας των διαφόρων χωρών μελών της ΔΟΘ, σε ό,τι αφορά τη β-θαλασσαιμία και την α-θαλασσαιμία, τη HbE και τη δρεπανοκυτταρική ...

Read More »

Κέντρα αναφοράς αιμοσφαιρινοπαθειών

Αυτό είναι ένα από τα νέα έργα της ΔΟΘ για το έτος 2013 και σκοπός του είναι να αναπτύξει μια βάση δεδομένων των Κέντρων αναφοράς αιμοσφαιρινοπαθειών σε ολόκληρο τον κόσμο, χαρτογραφώντας τις υπηρεσίες που παρέχουν στους ασθενείς. Το έργο αυτό αποτελεί διεύρυνση των πληροφοριών που συλλέχθηκαν στο πλαίσιο του έργου ...

Read More »