News
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wAIHA | Fostamatinib May Induce Durable Hemoglobin Response in Subset of Patients
Interestingly, this positive outcome was predominantly observed among participants hailing from North America, Australia, or Western Europe, while those from Eastern Europe didn’t show the same results. The current treatment…
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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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