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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
News

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?
Clinical News

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
Clinical News

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…
EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review
Clinical News

EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review

Τhe Chinese biotechnology company EdiGene announced yesterday that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug…
TIF At The NORBS Online Regional Conference For Rare Diseases – 23 0ctober 2020
News

TIF At The NORBS Online Regional Conference For Rare Diseases – 23 0ctober 2020

Supporting Serbia and other Eastern European countries in their efforts to upgrade holistic care services for patients with rare diseases, including thalassemia, TIF’s Executive Director, Dr Androulla Eleftheriou, participated  on…
Roche and Atea Pharmaceuticals Link Up To Co-Develop Remdesivir Rival Drug For COVID-19
Clinical News

Roche and Atea Pharmaceuticals Link Up To Co-Develop Remdesivir Rival Drug For COVID-19

The two companies will jointly develop, manufacture and distribute AT-527, Atea’s investigational oral antiviral for the potential treatment of COVID-19. According to Roche, AT-527 blocks the viral RNA polymerase enzyme…
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