First Sickle Cell Gene Therapy Patient Discharged After Landmark Treatment
核心洞察
Kendric Cromer, the first patient to receive the newly approved gene therapy for sickle cell disease (搜索), has been discharged from Children’s National Hospital after 44 days of treatment.
The FDA approved two gene therapies in December 2023, offering a potential permanent alteration of the genetic mutation causing sickle cell disease (搜索).
These therapies, developed by Bluebird Bio and Vertex Pharmaceuticals, provide a chance for patients to live free from the burdens of the disease, which affects approximately 100,000 people in the U.S.
Kendric Cromer, a 12-year-old, has become the first patient to be discharged from the hospital after receiving the groundbreaking gene therapy for sickle cell disease (搜索). Cromer had been undergoing treatment at the Children’s National Hospital in Washington, D.C., for 44 days before his discharge on Monday, marking a significant milestone in the treatment of this genetic disorder.
Until December 2023, individuals affected by sickle cell disease (搜索), which is caused by a genetic mutation in haemoglobin and impacts approximately 100,000 people in the U.S., had limited treatment options. The U.S. Food and Drug Administration (FDA) approved two revolutionary gene therapies developed by Bluebird Bio and Vertex Pharmaceuticals, offering a new outlook for patients.
These gene therapies aim to permanently alter the genetic mutation responsible for sickle cell disease (搜索), potentially freeing patients from the lifelong burdens of the condition. Cromer reflected on the impact of the disease on his childhood, stating, “I thought I would have sickle cell for the rest of my life.”
Cromer's parents highlighted the difficulties of the treatment process, noting that despite extensive consultations with doctors and thorough research, they were not fully prepared for the emotional and physical demands it would place on their family. “We knew it would be hard, but it was harder than we could have imagined,” his mother said.