Rare Anaemias
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News
wAIHA | Sovleplenib Demonstrates Positive Phase III Clinical Results
wAIHA is a rare autoimmune disorder in which the immune system produces antibodies that mistakenly target and destroy red blood cells, leading to anemia and significant clinical burden. The…
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SICKLE CELL DISEASE | Alzheimer’s Drug Demonstrates Potential Therapeutic Benefit
The study explores the potential of memantine, a medication that has been safely used for many years in the treatment of Alzheimer’s disease. Researchers investigated whether this well-known, affordable drug…
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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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SICKLE CELL DISEASE | FDA Grants Orphan Drug Designation to AND017
Announced by KIND Pharmaceuticals on October 25, 2024, ODD is granted to a drug or biological product to prevent, diagnose, or treat a rare disease or condition that affects fewer…
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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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APLASTIC ANEMIA | Hetrombopag Achieves Significant Hematological Responses in Newly Diagnosed Patients
In this study, newly diagnosed patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) were initially treated with either a combination of cyclosporin A (CsA) and hetrombopag (HETROM+CsA group) or CsA alone…
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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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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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