Rare Anaemias
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News
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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News
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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News
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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News
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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News
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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News
CLINICAL RESULTS | Roche Reports Positive Data For Crovalimab As Rare Blood Disease Treatment
PNH causes a patient’s red blood cells to break apart, resulting in a range of debilitating symptoms such as anaemia, fatigue, blood clots and kidney disease. The treatment is for…
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News
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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