Patients

EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
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EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia

The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia. This marks the first…
Just Updated: TIF’s Vaccinations & Therapeutic Drugs for COVID-19 Guide
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Just Updated: TIF’s Vaccinations & Therapeutic Drugs for COVID-19 Guide

Everything you need to know about the different COVID-19 vaccines, both already available and developing, in one place! Discover our newly updated Guide  »Vaccinations & Therapeutic Drugs for COVID-19 », containing…
COVID-19 Vaccines and Haemoglobin Disorders: The Latest Position Statement of the Thalassaemia International Federation
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COVID-19 Vaccines and Haemoglobin Disorders: The Latest Position Statement of the Thalassaemia International Federation

In view of the plans made by national health authorities in every country across the world for the widespread vaccination of individuals against the SARS-CoV-2 virus, the Thalassaemia International Federation…
TIF Guidelines For The Management Of Transfusion Dependent Thalassaemia 4th Edition Arriving Soon!
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TIF Guidelines For The Management Of Transfusion Dependent Thalassaemia 4th Edition Arriving Soon!

TIF, its Board of Directors, and International Scientific Advisory Board proudly announce that the 4th edition of the Federation’s most acclaimed and sought-after publication, TIF Guidelines For The Management of…
TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020
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TIF European Thalassaemia & Sickle Cell Disease Symposium To Take Place On 11-13 December 2020

TIF is proudly organising the European Thalassaemia & Sickle Cell Disease Symposium, a joint patients’ and healthcare professionals’ three-day educational event which will take place online on 11 – 13 December…
Virtual Conference on Alpha Thalassaemia Major By UCSF on 8-9 January 2021
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Virtual Conference on Alpha Thalassaemia Major By UCSF on 8-9 January 2021

The Center for Maternal-Fetal Precision Medicine (UCSF) will host a 2-day online international conference on Alpha Thalassaemia Major, which was once considered universally fatal in utero, on 8-9 January 2021. …
PK Deficiency Resources Now Available On TIF’s Website
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PK Deficiency Resources Now Available On TIF’s Website

Recognizing the similarities of thalassaemia with other rare anaemias, particularly those affecting red blood cells, (e.g. PKD, SCD), and noting the absence of disease-specific united internationally coordinated patient organizations to…
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
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What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?

There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in…
EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
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EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients

The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
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