RAIN
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News
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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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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REGULATORY NEWS | EMA Accepts Rocket Pharma’s Gene Therapy Application for Fanconi Anemia
RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. Mutations in FANCA hinder DNA repair and occur…
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EVENT | 1st RAIN Summit for Patients with Rare Anaemias | 18-19 May 2024
The workshop will present an excellent opportunity for patients to exchange knowledge on the latest scientific advances for Rare Anaemias, and share concerns and challenges. A unique occasion for a…
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REGULATORY NEWS | EU Backs Revolutionary Oral Monotherapy for Adult PNH Patients
The decision was hailed as a first step toward enabling patient access in European Union countries following a March 21 meeting of the Committee for Medicinal Products for Human Use…
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SPOTLIGHT | RAIN Endorses the First International Guidelines for Pyruvate Kinase Deficiency
Pyruvate kinase deficiency (PKD) is the most common cause of chronic congenital non-spherocytic haemolytic anaemia, impacting between one in 100,000 to one in 300,000 individuals globally. Those with PKD suffer…
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