RAIN
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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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PUBLICATIONS | Nutrition Guide for Patients with Thalassemia & PKD
Nutrition holds a significant position in the lives of individuals afflicted with thalassaemia and other hereditary anaemias. As part of its enduring commitment to generating guidelines and educational materials for…
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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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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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GENE THERAPY | FDA Decision on Lovo-cel for SCD Now Expected by Year’s End
The regulatory agency has agreed to review an application from lovo-cel’s developer, Bluebird Bio, requesting the gene therapy’s approval for SCD patients ages 12 and older. Moreover, the FDA has given the application priority…
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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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