NHS Scotland has approved a one-time CRISPR gene therapy for severe sickle cell disease, offering durable reduction in vaso-occlusive crises for eligible patients.

Two Pee Dee siblings are officially cured of sickle cell disease after more than a decade of living with the genetic blood ...

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 ...

Key HighlightsMotixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies73% ...

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 ...

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, doctors said he's essentially "cured" thanks to gene therapy treatment at ...

At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive director of the nonprofit patient advocacy group the Sickle ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...

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 ...

What you need to know: Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease A biotechnology company in Uganda has developed a new ...