Clinical News
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As survival increases in β-thalassemia, renal complications emerge
In a scientific paper recently published in Hematology, researchers discussed the most common pathophysiologic and clinical manifestations of renal disease in patients with beta thalassaemia. “In recent years, the…
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Biotech companies issue first joint declaration on human gene editing
Companies involved in human gene editing have entered the ethical debate about the controversial technology, issuing a joint declaration that DNA must not be altered in a way that passes…
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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…
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Oxbryta Increased Haemoglobin And Reduced Haemolysis in Adults and Adolescents With SCD
Global Blood Therapeutics, Inc. announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study of Oxbryta® (voxelotor) tablets in patients with sickle cell disease…
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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…
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PYRUVATE KINASE DEFICIENCY | FDA Approves First Drug For The Rare Inherited Anemia
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…
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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…
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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…
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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…
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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…
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