Sickle Cell Disease
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News
SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017
Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer…
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Clinical News
WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and…
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Scientific News
Stroke Risk Rises in People with Sickle Cell Disease Despite Treatment Guidelines
A new study reveals that stroke rates are continuing to rise among both adults and children living with sickle cell disease (SCD), despite established standards of care aimed at reducing…
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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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