Gene Therapy for Deadly Blood Disorder Restores Function in Patients' Cells

UCLA researchers have made strides in developing a stem cell gene therapy for alpha thalassemia major, a severe, inherited ...

Gene Therapy Could End Lifelong Transfusions For Rare Blood Disorder

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

New gene therapy now administered as Sickle Cell Anemia treatment in St. Louis Children’s Hospital

According to the CDC, nearly 100,000 Americans have Sickle Cell Anemia, or 1 in every 265 African American births.
Nature

Huntington’s disease treated for first time using gene therapy

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.
WPLG Local 10

South Florida teen becomes first in area to receive FDA-approved gene therapy for sickle cell

BROWARD COUNTY, Fla. — A South Florida family is happy their son no longer lives with chronic pain. Plus, he’s the first in the area to go through FDA-approved gene therapy. Caden Major had been ...
Morningstar

Beyond Gene Therapy: Pyruvate Kinase Activators (Agios, Novo Nordisk) Offer a New Route to Address Hemoglobin, VOCs, and Transfusion Burden in Sickle Cell Disease

EXTON, PA, Aug. 06, 2025 (GLOBE NEWSWIRE) -- According to the latest findings from Spherix Global Insights’ syndicated Market Dynamix™ service, the US sickle cell disease (SCD) market is approaching a ...

Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

A one-time experimental treatment shows unprecedented success in slowing Huntington's disease, according to UCL researchers.

U.K. gene therapy trial effectively treats Huntington's disease

Researchers at University College London reported success in treating Huntington's disease with gene therapy, which is the ...