Bleeding Disorders
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Democratic Lawmakers Press Pfizer on Chemotherapy Drug Shortages
The company, which said in May that it was working to assuage critical shortages of methotrexate, carboplatin and cisplatin, is being asked to...
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CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval
Ten weeks ahead of target date, FDA greenlights this innovative treatment for a rare blood disorder.
Conference Coverage
Sickle Cell CRISPR Gene Therapy May Offer Patients ‘Functional Cure’
Patients with severe sickle cell disease aged 12 and older could benefit greatly from two newly approved gene-editing treatments.
Conference Coverage
SCD mortality rates improved for Black patients in 2010s
The average age at death rose 4 years during that decade, but the transition from pediatric to adult care remains a major danger zone.
Conference Coverage
Sickle Cell: Good Outcomes for Haploidentical Transplants
Overall 2-year survival was 95% in a small, “robust” phase-2 trial of children and adults with sickle cell disease.
Conference Coverage
Sickle Cell Gene Therapy ‘Truly Transformative’
New research on a just-approved gene therapy for sickle cell disease shows the efficacy of a single infusion of lovo-cel.
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FDA approves first 2 gene-editing therapies for sickle cell
These expensive “milestone treatments” greenlighted by the FDA are the first gene-based therapies for sickle cell disease.
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FDA greenlights iptacopan to treat paroxysmal nocturnal hemoglobinuria
The first oral monotherapy for a rare chronic blood disorder, paroxysmal nocturnal hemoglobinuria, has been approved by the FDA.
Conference Coverage
Hematology is in the Brodsky family’s blood
Three generations of Brodsky men have forged careers in the same medical field.
Conference Coverage
ASH 2023: Equity, Sickle Cell, and Real-Life Outcomes
Leaders of the American Society of Hematology preview hot topics and presentations on tap at their 2023 annual meeting.
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FDA approves first tx for rare, deadly clotting disorder
First drug to treat TTP. a rare, lethal blood clotting disorder, has been greenlighted by the FDA.