Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...

At the Cleveland Clinic, doctors are using a one-time gene-editing therapy that alters a patient’s own blood-forming stem cells to correct the genetic mutation responsible for the disease. Physicians ...

Fifteen-year-old Jayden Wilsey of Falmouth is the first person at Boston Children's Hospital to receive human gene therapy for sickle cell disease, following approval of the therapy in 2024 by the U.S ...

Sickle cell disease is a devastating, inherited blood disorder that affects an estimated eight million people worldwide and ...

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD). The regulator said ...

Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of ...

Exagamglogene autotemcel (exa-cel) significantly improved HRQOL in severe SCD patients, eliminating severe VOCs and hospitalizations in most cases. The CLIMB SCD-121 trial showed sustained ...

LOS ANGELES (KABC) -- It's been more than a month since the FDA approved two milestone gene-editing treatments for sickle cell disease, but lining up patients for these therapies will be a challenge ...

Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA ...

CHICAGO (WLS) -- Sickle cell anemia disproportionately affects Black people, causing severe pain, potential organ damage and in some cases, death. Many times, only blood transfusions can provide ...

Public health experts have emphasised the urgent need for Nigeria to adopt life-changing gene therapy for the treatment of sickle cell disease. Official data shows that approximately 4 million ...