Catherine Skari

Ms Katia Pelides

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CLINICAL NEWS | Agios Meets Clinical Proof-of-Concept in Anaemia Treatment for Adults with Lower-Risk MDS

On Monday, the specialized disease company announced the successful achievement of “proof-of-concept” in a Phase 2a trial involving AG-946, a…

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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…

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NEW VIDEO | Jo’s Story With β-Thalassaemia

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BREAKING NEW GROUND | The First-Ever Guidelines for Managing α-Thalassaemia

The Thalassaemia International Federation (TIF) is proud to present the first international-level guidelines for the management of a-thalassaemia, a comprehensive…

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Association of Osteoporosis and Sarcopenia with Fracture Risk in Transfusion-Dependent Thalassemia

Patients with transfusion-dependent thalassaemia (TDT) have an increased risk of osteoporosis and fractures. They also have several potential factors associated…

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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…

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Splenectomy Significantly Associated with Thrombosis but Not with Pulmonary Hypertension in Patients with Transfusion-Dependent Thalassemia: A Meta-analysis of Observational Studies

Introduction: Thromboembolism (TE) and pulmonary hypertension (PH) constitute frequently occurring complications in patients with transfusion-dependent thalassemia and have been associated…

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Deferiprone vs Deferoxamine for Transfusional Iron Overload in Sickle Cell Disease and Other Anemias: Pediatric Subgroup Analysis of the Randomized, Open-label FIRST Study

Background: Children with sickle cell disease (SCD) who are chronically transfused often, require iron chelation therapy. There are limited data…

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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | LDH Improvements Lead to Better Outcomes in Fatigue and QoL

PNH is a rare anaemia known for causing blood cell destruction, which can be shown by LDH levels. The study…

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