Researchers at the Eye Genetics Research Unit at Children's Medical Research Institute (CMRI) are the first in the world to ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational gene therapy showed sustained clinical efficacy and safety at a median ...

The Cincinnati Children’s Center has expanded its capacity to manufacture cellular products for rare diseases.

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

Sharonville, Ohio, celebrates the opening of the new Cincinnati Children's Applied Gene and Cell Therapy Center.

Penn and CHOP researchers will utilize the new protocol to distribute customizable drugs that target specific genetic disorders.

TAMPA, Fla. (Ivanhoe Newswire) - It’s a devastating diagnosis that changes a child’s life, and their family’s, forever. Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that ...

A Greer couple is hosting a fundraiser to support a costly gene editing procedure for their two children, who suffer from a ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment ...

Gene therapy has allowed several children born with inherited deafness to hear. A small study published Wednesday documents significantly restored hearing in five of six kids treated in China. On ...

The STEER study showed significant motor function improvement in children with SMA using intrathecal onasemnogene abeparvovec. The primary efficacy endpoint was met with a 2.39-point improvement in ...