Articles related to SICKLE CELL DISEASE (SCD)

The FDA expanded approval of the CRISPR-based gene therapy Casgevy to include patients as young as 2 years with sickle cell disease or transfusion-dependent beta thalassemia. The decision was supported by pediatric phase 3 data demonstrating sustained reductions in disease burden.

Prior to CDC’s Sickle Cell Data Collection program, the actual number of Americans living with sickle cell disease was unknown, with current estimates of 100,000 patients based on systematic data collection efforts now at risk.

Hemoglobin disorders represent the single largest category of genetic disorders in the United States after chromosomal disorders, yet there is no systematic approach to managing them despite excellent guidelines from NHLBI, ASH, and NASCC.

The comparative modeling analysis of gene therapy for sickle cell disease underscores its potential cost-effectiveness and value in enhancing patient quality of life. With estimated ICERs significantly varying under different perspectives, the findings advocate for a nuanced approach to assessing novel therapies within the broader healthcare landscape.

Gain actionable insights into the diagnosis and management of nonmalignant blood disorders at Mayo Clinic’s Classical Hematology 2023 conference. Elevate your practice with evidence-based strategies from leading experts.

This study provides new insights into using electronic health records as an efficient tool for predicting survival outcomes in SCA treatment, highlighting the significant role of hydroxyurea in lowering mortality rates.