RAIN
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News
SICKLE CELL DISEASE | Inflammation during Menstrual Cycle May Trigger Pain Crises
SCD is a genetic disorder in which red blood cells can become abnormally shaped (“sickle”), leading to blockages in blood vessels, tissue damage, and intense pain known as vaso-occlusive events…
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SEVERE APLASTIC ANAEMIA | Stem Cell Transplant Opens New Possibilities
Severe aplastic anaemia is a rare but serious disorder in which the bone marrow fails to produce enough red and white blood cells or platelets. This results in fatigue, infections,…
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SICKLE CELL DISEASE | Limited Progress in Sickle Cell Disease Highlights Barriers to Treatment Access
According to the findings, there has been limited improvement in outcomes for SCD patients, in large part due to barriers in accessing first-line therapies such as hydroxyurea. Although several new…
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SICKLE CELL DISEASE | Intranasal Fentanyl Shows Promise for Rapid Pain Relief in Adults
Fentanyl is a powerful painkiller that has already been widely used in children with SCD and in adults with cancer or chronic pain. Researchers now believe its use in adults…
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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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