Sickle Cell Disease
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Scientific Literature
An International Learning Collaborative Phase 2 Trial for Haploidentical Bone Marrow Transplant in Sickle Cell Disease
In the setting of a learning collaborative, we conducted an international multicentre phase 2 clinical trial testing the hypothesis that non-myeloablative related haploidentical BMT with thiotepa and post-transplant cyclophosphamide (PTCy)…
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News
ADVOCACY IN ACTION | Highlights from the TIF Meeting with Greek Health Minister, Adonis Georgiadis
A productive meeting between the Greek Minister of Health, Mr. Adonis Georgiades, and a Thalassaemia International Federation (TIF) delegation took place on February 7, 2024. The discussions centered on longstanding…
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Scientific Literature
LentiGlobin Administration to Sickle Cell Disease Patients: Effect on Serum Markers and Vaso-Occlusive Crisis
LentiGlobin, an innovative gene therapy, introduces a modified beta-globin gene that yields an anti-sickling hemoglobin variant. It boosts total hemoglobin levels, mitigates hemolysis, curtails inflammation, and addresses iron overload by…
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News
SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
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Scientific News
CASGEVY | 1st Gene-Editing Therapy for Thalassaemia and SCD Approved in UK
Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The therapy, developed by…
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Scientific Literature
Lung Function Decline in Children with Sickle Cell Disease Treated with Hydroxyurea
Sickle cell disease (SCD), the most common form of an inherited hematological disorder, is caused by a genetic variant that leads to the synthesis of an abnormal haemoglobin, HbS. Deoxygenated…
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Scientific Literature
Impact of Imatinib on Reducing the Painful Crisis in Patients with Sickle Cell Disease
Introduction: Sickle cell disease (SCD) is a common hemoglobinopathy worldwide that causes painful crises and hospitalization of patients. These attacks decrease survival and cause chronic end-organ damage in these patients. Hypothesis: For this reason,…
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Scientific Literature
Modelling the Public Health Impact of Voxelotor in the Management of Sickle Cell Disease in France
Sickle cell disease (SCD) is an inherited blood disorder in which sickle hemoglobin (HbS) polymerizes, leading to red blood cell sickling and chronic hemolytic anemia, vaso-occlusive crises, and end-organ damage…
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Scientific Literature
Management of the Sickle Cell Trait: An Opinion by Expert Panel Members
The number of individuals with the sickle cell trait exceeds 300 million worldwide, making sickle cell disease one of the most common monogenetic diseases globally. Because of the high frequency…
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News
DRUGS | EU Pulls Authorization of Adakveo for Sickle Cell Disease
The decision comes after the Committee on Medicinal Products for Human Use (CHMP) recommended in May 2023 to pull the approval following a review of additional Phase III data. The EC’s decision…
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