RAIN
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News
PREGNANCY | Study Results Indicate AIHA in Pregnant Women Is Manageable
Among these patients, however, close monitoring, the administration of therapy when needed, and the awareness of potential complications, both for the mother and fetus, are essential for maximizing the odds…
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EHA2023 | MASP-3 Inhibitor Demonstrates Clinical Efficacy in Paroxysmal Nocturnal Hemoglobinuria
OMS906 led to transfusion independence in the majority of patients with PNH enrolled so far on the trial (n = 10). PNH, a rare and life-threatening blood disorder, is characterized…
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EVENT | Rare Anaemias International Network (RAIN) Members Meeting – 16 June 2023
The members meeting was open to all RAIN members and their individual networks. This Meeting sought to: provide an update and overview of the RAIN Steering Committee and Scientific Advisory…
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NEW TREATMENTS | Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies
That is the major finding of a newly published review that examined how a better understanding in the scientific and molecular underpinnings of the disease has translated into potential new…
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CLINICAL NEWS | Novartis Releases Data for Potentially “Practice-Changing” PNH Medicine
The results, announced via news release, were presented at the 49th Annual Meeting of the European Society for Blood and Marrow Transplantation in Paris. “The APPOINT-PNH results are consistent with…
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PK DEFICIENCY | New Episode of ‘Just Listen’ Podcast Out Now
Check out the latest episode of ‘Just Listen: Voices of PK Deficiency’ podcast, where host Dr Rachael Grace converses with Dr Matthew Heeney, a haematologist whose research is focused on the…
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APLASTIC ANEMIA | Eltrombopag May Increase Complete Response Rate Among Pediatric Patients
The standards of care for aplastic anemia are allogeneic hematopoietic stem cell transplantation (HSCT) or immunosuppression with horse antithymocyte globulin and cyclosporine. Early data suggest that adding ELTR to immunosuppression…
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News
G6PD | Common Enzyme Deficiency Linked With COVID Severity
In Black men under 65, for example, those with the common enzyme deficiency had a roughly 1.5-fold higher risk for severe illness (OR 1.47, 95% CI 1.03-2.09), reported Lavannya Pandit,…
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NEW REVIEW | Treatment Landscape Of Rare Anaemias Is Shifting Rapidly
Writing in Frontiers in Medicine, the authors began by reviewing congenital and acquired forms of anaemia. Those include sickle cell disease (SCD) and hemolytic anemias (CHAs), both congenital, and autoimmune hemolytic…
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SICKLE CELL DISEASE | Hydroxyurea Use Linked To Higher Rates Of Spleen Removal
The study’s findings also indicated that SCD children treated with hydroxyurea underwent splenectomy surgery at a significantly younger age compared with those not given the oral medication. That result came…
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