Скачать или смотреть Gene Therapy for Beta Thalassemia: Rahemeen’s Story

A curative gene therapy that CHOP helped develop transformed Rahemeen’s life. https://www.chop.edu/centers-programs...

Rahemeen was born with beta thalassemia, an inherited blood disorder where the body is unable to make enough hemoglobin, the substance that carries oxygen throughout the body. As a result, a patient needs to get blood transfusions every few weeks for their entire life.

In a game-changing breakthrough for patients with this condition, and after rigorous clinical trials that took place at Children’s Hospital of Philadelphia (CHOP) and other sites, the Food and Drug Administration (FDA) approved a gene therapy for the treatment of beta thalassemia. CHOP was the first Qualified Treatment Center offering the treatment, called Zynteglo.

This video tells the story of Rahemeen, the first patient at Children's Hospital of Philadelphia who received the therapy following its FDA approval.