Sickle Cell Disease
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Scientific Literature
Reduction in Vaso-Occlusive Events Following Hematopoietic Stem Cell Transplantation in Patients with Sickle Cell Disease
Hematopoietic stem cell transplantation (HSCT) is potentially curative for patients with sickle cell disease (SCD). Patients with stable donor engraftment after allogeneic HSCT generally do not experience SCD-related complications; however,…
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Scientific Literature
Association of Sickle Cell Disease With Severe Maternal Morbidity
Objective: To evaluate the association between sickle cell disease (SCD) and severe maternal morbidity (SMM) in a contemporary cohort of deliveries by non-Hispanic Black people. Methods: We retrospectively examined SMM…
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News
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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News
CLINICAL UPDATE | Graphite’s Hopes For Sickle Cell Cure Blunted After First Patient Dosed Experiences Serious Event
The company cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) as part of a phase 1/2 trial for SCD. The event is likely to…
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News
DRUGS | EMA Starts Pilot To Collect Information On Ongoing/Potential Shortages
To help address this issue, the European Medicines Agency (EMA) is starting a pilot to collect information on ongoing or potential shortages, according to the following criteria: The shortage…
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News
CDC | Many Children With Sickle Cell Anaemia In The US Not Receiving Lifesaving Screening And Treatment
According to a recent CDC study, fewer than half children with the disease in the US get the necessary screening, and only about half or fewer get treatment with hydroxyurea…
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Clinical News
SAFER BLOOD | EU Patients Have Begun Receiving Transfusions With Hemanext ONE® RBC Processing & Storage System
Doctors have begun transfusing patients suffering from hematological malignancies using RBCs processed and stored with the Hemanext ONE® system, as part of a post-market clinical study in Bergen, Norway. This is…
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Scientific Literature
Precision Medicine and Sickle Cell Disease
Sickle cell disease (SCD) is characterized by variable clinical outcomes, with some patients suffering life-threatening complications during childhood, and others living relatively symptom-free into old age. Because of this variability,…
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News
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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News
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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