Catherine Skari

NEWS | ICER Releases Draft Evidence Report on Treatment for Paroxysmal Nocturnal Hemoglobinuria

This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document…

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PHARMA NEWS | FDA Approves Iptacopan for Paroxysmal Nocturnal Hemoglobinuria

Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing comprehensive…

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NEW WEBINAR | Empowering Patient Leaders: Understanding EU Health Policies

📌 Webinar Title: ‘‘Empowering Patient Leaders: Understanding EU Health Policies’’ 📌 Date & Time: 18 December 2023, Monday, at 14:00…

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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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