News
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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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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 should not be interpreted to be ICER’s final conclusions. Register…
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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 control of red blood cell (RBC) destruction within and outside…
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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 EET 📌 Registration: https://forms.office.com/e/nTz4yTd9NA In this webinar, we aim to…
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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 pyruvate kinase (PK) activator aimed at treating anemia in patients…
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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 awarded the Nobel Prize in 2020. The therapy, developed by…
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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 and indispensable resource for health professionals, researchers and all those…
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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 compared two drugs, eculizumab and ravulizumab, to see how they…
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TIF’s NEW e-COURSE | Enhance Your Understanding of EU Health Policy
To address this need, the Thalassaemia International Federation (TIF) has launched an e-course titled ‘EU Health Policy Course’, which offers a comprehensive and up-to-the-minute overview of EU health-related policies. The…
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