Healthcare Professionals

Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical News

Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy

The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
News

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…
TIF Guidelines For The Management Of Transfusion Dependent Thalassaemia 4th Edition Arriving Soon!
News

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
News

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
News

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…
eThalED Webinar Series: Current Standard Care & New Advances in Iron Chelation
eThalED Webinars

eThalED Webinar Series: Current Standard Care & New Advances in Iron Chelation

TIF´s last webinar for the eThalED series for Medical Specialists on October 16, 2020, featured Dr Farrukh Shah, Consultant Haematologist, Whittington Hospital, London, UK, who reviewed all the current practices…
Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
Clinical News

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Fulcrum Therapeutics, Inc.  a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and…
Schaltfläche "Zurück zum Anfang"