SCD disease burden heavy in US, despite emerging gene therapies

SCD disease burden heavy in US, despite emerging gene therapies

In the U.S., having sickle cell disease (SCD) continues to place a heavy clinical burden on patients, who often require multiple medications, frequent blood transfusions, and the use of opioids to control pain. Most individuals with SCD report high barriers to healthcare, with poor access to treatments and little effective pain management as major issues. Meanwhile, though emerging treatments such as gene therapy are generating interest among do…