Rare Anaemias
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News
CLINICAL NEWS | FDA Approves Luspatercept for First-line Treatment of Anemia in Lower-Risk MDS
The approval was based on interim results from the pivotal phase 3 COMMANDS trial (ClinicalTrials.gov Identifier: NCT03682536) that evaluated the efficacy and safety of luspatercept in 356 patients with anemia due to international…
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COLD AGGLUTININ DISEASE | Enjaymo Improved Life Quality in Patients for 2.5 Years
“Enjaymo provides sustained and durable treatment benefits in chronic CAD, including a continued meaningful impact on patient quality of life in the long term,” the researchers wrote. However, these benefits…
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CLINICAL TRIALS | Positive Results Found With Transplant For Patients With Aplastic Anemia
The findings were reported in the journal Blood. The trial’s cohort included 35% of patients from underrepresented racial and/or ethnic groups, suggesting that this BMT method for patients without a…
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PUBLICATION | Travel Guidance for Patients and Families
Welcome to ‘Travel Guidance for Patients & Families’, on the newest TIF’s publications! This practical handbook is intended specifically for travelers with thalassaemia, sickle cell disease, and rare anaemias. Our…
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CALL TO ALL PATIENTS | Your Voice Matters in Research!
Today, we invite you to be an active participant in ensuring that research that takes place is aligned with your specific requirements and challenges. Your firsthand experiences as patients offer…
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EHA2023 | Crovalimab Appears Non-inferior to Eculizumab in PNH, Data Shows
At the 2023 EHA Congress, researchers presented data from the ongoing COMMODORE 2 study, which was designed to determine whether crovalimab is non-inferior to eculizumab for multiple clinical endpoints among…
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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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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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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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