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Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood ...

– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support ....

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Bristol Myers Squibb's First Disclosures and New Data at ASH 2023 Highlight Company's Leadership and Progress in Cell Therapy, Targeted Protein Degradation and Novel Approaches in Hematology -November 02, 2023 at 11:35 am EDT

First presentation of results from primary analysis of Phase 2 TRANSCEND FL study evaluating second-line treatment with Breyanzi® in relapsed or refractory follicular lymphoma demonstrate its. ....

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Teenage Patient Gives Back to Columbia Clinic

Teenage Patient Gives Back to Columbia Clinic
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Agios Recognizes 14th Annual Rare Disease Day and Raises Awareness of Unmet Needs in Rare ...

Agios Recognizes 14th Annual Rare Disease Day and Raises Awareness of Unmet Needs in Rare ...
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CRISPR technology to cure sickle cell disease at UIC

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University of Illinois Chicago is one of the U.S. sites participating in clinical trials to cure severe red blood congenital diseases such as sickle cell anemia or Thalassemia by safely modifying the DNA of patients blood cells.
The first cases treated with this approach were recently published in an article co-authored by Dr. Damiano Rondelli, the Michael Reese Professor of Hematology at the UIC College of Medicine. The article reports two patients have been cured of beta thalassemia and sickle cell disease after their own genes were edited with CRISPR-Cas9 technology. The two researchers who invented this technology received the Nobel Prize in Chemistry in 2020. ....

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