Κλινικά Νέα
Oxbryta Gets Marketing Authorisation In The UK For Patients With Sickle Cell Disease
3 εβδομάδες ago
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…
BLOOD TRANSFUSION | Hemanext Begins Rollout of Hemanext ONE® RBC Processing And Storage System In Europe
14 Ιουλίου, 2022
BLOOD TRANSFUSION | Hemanext Begins Rollout of Hemanext ONE® RBC Processing And Storage System In Europe
Physicians in Norway have initiated recruitment of chronically transfused patients with haematological malignancies, including myelodysplastic syndrome (MDS), to receive transfusions using RBCs processed and stored with the Hemanext ONE® system.…
SCIENTIFIC NEWS | Human Trials Of Universal Flu And COVID-19 Vaccines Begin
12 Ιουλίου, 2022
SCIENTIFIC NEWS | Human Trials Of Universal Flu And COVID-19 Vaccines Begin
The National Institutes of Health’s (NIH) work on a universal flu vaccine is already well advanced, with human trials beginning now. The new formulation, called BPL-1357, contains a variety…
Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency
18 Φεβρουαρίου, 2022
Mitapivat Receives FDA Approval For Haemolytic Anaemia From Pyruvate Kinase Deficiency
The US Food and Drug Administration (FDA) has announced the approval of Mitapivat (PYRUKYND®) tablets as treatment for haemolytic anemia in adults with pyruvate kinase (PK) deficiency. The oral PK…
What’s New In Clinical Trials For Thalassaemia/Sickle Cell Disease Drugs And Therapies?
13 Ιανουαρίου, 2022
What’s New In Clinical Trials For Thalassaemia/Sickle Cell Disease Drugs And Therapies?
Many pharmaceutical companies are making remarkable progress in their research into candidate treatments for these diseases and 2022 is expected to be yet another eventful year for haemoglobin disorders! Check…
SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU
7 Ιανουαρίου, 2022
SCD Drug Oxbryta Receives CHMP Positive Opinion For Approval In The EU
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending marketing authorization for Oxbryta® (voxelotor) tablets for the treatment of hemolytic…
FDA Grants Priority Review to Luspatercept-aamt for Anemia in Adults With NTD β-Thalassaemia
6 Δεκεμβρίου, 2021
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…
La Repubblica: Gene Editing Cured Two Siblings With Thalassaemia In Italy
10 Νοεμβρίου, 2021
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…
TIF Releases New Scientific Bulletin To Highlight Research On Haemoglobin Disorders
27 Οκτωβρίου, 2021
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…
UK & Wales: NICE Recommends First Treatment In Two Decades For Sickle Cell Disease
2 Οκτωβρίου, 2021
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…