Clinical News
-
A milestone in the treatment of b-thalassaemia – The 1st gene therapy for Transfusion Dependent β-Thalassaemia wins approval in Europe
Zynteglo, Bluebird Bio’s ex vivo gene therapy for β-thalassaemia, got a green light from the EMA for conditional marketing authorization making it the 1st gene therapy for patients with…
Read More » -
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency (EMA) has validated the Type II variation…
Read More » -
La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy
Cured of thalassemia, thanks to gene editing. According to a recent news report published in the Italian newspaper La Repubblica, Erika and Emanuele Guarini, two siblings from Pistoia, Tuscany, aged…
Read More » -
From labs to shelves: EMA’s new booklet explains how drugs are developed and marketed in the EU
The European Medicines Agency (EMA) has published a booklet describing the journey of a medicine for human use authorised through EMA, from initial research to discussions on patient access…
Read More » -
Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease
Voxelotor, an oral treatment taken once daily, is the first medicine authorized in Great Britain that directly inhibits sickle haemoglobin (HbS) polymerization, the molecular basis of sickling and destruction of…
Read More » -
-
TIF Releases New Scientific Bulletin To Highlight Research On Haemoglobin Disorders
A key objective of the Thalassaemia International Federation is to inform and update regularly the global haemoglobinopathies family, both patients and physicians, on the progress and new developments that arise…
Read More » -
-
CLINICAL ALERT: New study highlights ‘unsafe’ T2* MRI method being used for iron overload assessment in thousands of patients
New study highlights ‘unsafe’ T2* MRI method being used for iron overload assessment in thousands of patients. TIF became aware of the results from a new study, to be presented…
Read More » -
UK & Wales: NICE Recommends First Treatment In Two Decades For Sickle Cell Disease
For the first time in 20 years, a new therapy for sickle cell disease is to be made available on the UK National Health Service (NHS). Crizanlizumab (Adakveo) by Novartis…
Read More »