News
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REGULATORY APPROVAL | PiaSky Becomes the First Monthly Treatment for PNH in the EU
Paroxysmal Nocturnal Haemoglobinuria (PNH) is a rare and life-threatening blood condition where red blood cells are destroyed by the complement system – part of the innate immune system – causing…
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ACCESS TO INNOVATION | UK Greenlights Gene-Editing Therapy Casgevy for β-Thalassaemia Treatment
This development opens new treatment avenues for an estimated 460 patients aged 12 and above in England who rely on regular transfusions due to β-thalassaemia, leveraging innovative CRISPR gene-editing technology.…
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GLOBAL SURVEY | Understanding the Thalassaemia Community Health Literacy & Its Impact on Care
To improve the understanding of health literacy within the thalassemia community and how this may impact disease management and care, a global survey is being conducted by the Thalassemia Advocacy…
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JUST LISTEN | Voices of PK Deficiency Episode on the Impact of New International Standards
Host Dr. Rachael Grace is joined by Tamara Schryver of Thrive with Pyruvate Kinase Deficiency and Alejandra Watson from the PKD Foundation to shed light on these ground-breaking guidelines. Listeners…
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NEW APPROVAL | FDA Endorses Piasky for Paroxysmal Nocturnal Hemoglobinuria
Piasky, developed by Roche, is a monoclonal antibody that targets complement protein C5. Crovalimab binds to C5 to prevent its cleavage, thus preventing the formation of the membrane attack complex…
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CAD | Enjaymo Not Cost-Effective at Current Price, According to US Study
This conclusion was drawn from a cost-effectiveness study utilizing data from the Phase 3 CARDINAL clinical trial, which backed the treatment’s approval for CAD patients with a recent history of…
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SPOTLIGHT | TIF Wins Prestigious HemaSphere Award at EHA2024
The HemaSphere Awards were presented by the official journal of the European Hematology Association (EHA) during Europe’s most important scientific event in the field of Haematology, EHA2024 Hybrid Congress, held…
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NEW PUBLICATION | Nutrition in Thalassaemia: A Guide for Patients, Families, and Caregivers
The guide encompasses: An overview of the nutritional needs specific to people with thalassaemia Methods for nutritional assessment Detailed explanations of nutrients particularly important for thalassaemia patients Insights into how…
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