Sickle Cell Disease
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News
EMPOWERING NURSING PRACTICE IN HAEMOGLOBINOPATHIES | Discover the New TIF Guide
Nurses are the heart of haemoglobinopathy care. Their role spans far beyond routine tasks—encompassing complex clinical duties, patient advocacy, case management, emotional and psychosocial support, and education of both patients…
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Scientific News
WHO Issues First Global Guideline on Sickle Cell Disease in Pregnancy
Why This Matters High maternal risk: Pregnant women with SCD face a 4–11× greater risk of maternal mortality and elevated rates of pre-eclampsia, while their infants are at increased…
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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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Scientific 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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