Rare Anemias
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News
NEW STUDY | Pegcetacoplan Shows Potential in Managing CAD and wAIHA
This investigational therapy, which targets complement component C3, has demonstrated the ability to increase haemoglobin levels, reduce haemolysis, and improve fatigue scores, according to the study’s authors. Although not all…
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PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Patients May See Improved Outcomes with Ravulizumab
The findings showed that patients treated with ravulizumab demonstrated better adherence and persistence compared to those on other complement inhibitors. The ADVANTAGE study, a retrospective longitudinal analysis, included data from…
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Splenectomy as Treatment for Primary Warm Autoimmune Hemolytic Anemia (wAIHA)
Warm Autoimmune Hemolytic Anemia (wAIHA) occurs when the body’s immune system mistakenly produces antibodies that attack and destroy its own red blood cells. In this particular case, the patient presented…
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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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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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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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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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COLD AGGLUTININ DISEASE | Enjaymo Improved Life Quality in Patients for 2.5 Years
“Enjaymo provides sustained and durable treatment benefits in chronic CAD, including a continued meaningful impact on patient quality of life in the long term,” the researchers wrote. However, these benefits…
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COLD AGGLUTININ DISEASE | Enjaymo Improves Life Quality And Lessens Fatigue, According to Phase 3 Trial Results
These findings “further augment the primary efficacy outcomes of the placebo-controlled Phase 3 CADENZA study, demonstrating that in addition to providing improvements in hematologic [blood-related] parameters, treatment with [Enjaymo] also…
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