South Florida teen hopeful after new gene therapy treatment for sickle cell anemia

Caden Major was born with sickle cell disease – an inherited blood disorder affecting red blood cells. Now at 19 years old, ...
News On 6

New gene therapies offer hope for sickle cell patients

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 ...

Researchers say there is now a cure for sickle cell disease

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

Cape Cod teen to receive human gene therapy for sickle cell. He is one of the first.

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 ...
The American Journal of Managed Care

Gene Therapy Meaningfully Improves Quality of Life in Severe Sickle Cell Disease

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 ...
Medscape

NICE Backs CRISPR Gene Therapy for Severe Sickle Cell Disease

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending a CRISPR gene editing-based treatment for severe sickle cell disease (SCD).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

Ayrmid Reports Promising New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease

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 ...
ABC7

FDA-approved gene therapies bring hope for sickle cell patients, but high costs pose challenges

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 ...
ABC 7 Chicago

Sickle cell patient Braxton Hubbard feels 'amazing' after gene therapy treatment

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 ...
Morningstar

Gene Therapy Momentum Builds Among Hematologists in Sickle Cell Disease Management Despite Ongoing Access Barriers, According to Spherix Global Insights

Gene Therapy Momentum Builds Among Hematologists in Sickle Cell Disease Management Despite Ongoing Access Barriers, According to Spherix Global Insights Provided by GlobeNewswire May 1, 2025, 3:42:51 ...
The Nation Newspaper

Sickle cell: Experts urges adoption of gene therapy

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 ...