SCD
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NEW HOPE FOR THALASSAEMIA/SCD | UK Trial Of Lab Grown Red Blood Cells Begins
The Restore trial, led jointly by NHS Blood and Transplant and the University of Bristol, is studying the lifespan of the lab grown cells compared with infusions of standard red blood…
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SICKLE CELL DISEASE | African Health Ministers Launch Drive to Curb SCD’s Toll
This effort, backed by the African Regional Office of the World Health Organization (WHO), aims to curb SCD΄s toll in a region that has historically been hit hard by this…
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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…
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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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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…
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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…
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Global Blood Submits US Application For Expanded Use Of SCD Drug, Oxbryta, In Patients Aged 4-11 Years
Children with sickle cell disease (SCD) as young as age 4 could be prescribed Oxbryta (Voxelotor) if the U.S. Food and Drug Administration (FDA) approves a supplemental new drug application (sNDA) submitted by…
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Adakveo (crizanlizumab)
Update: 31 March 2023 Preliminary results from the ongoing global Phase III STAND study in sickle cell disease showed no difference between Adakveo and placebo in annual rates of…
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Oxbryta (voxelotor)
Update: 31 March 2023 Preliminary draft guidance from England’s HTA body NICE states the product is not an acceptable use of National Health Service resources. Previously the UK regulatory…
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