ATA-100 (AAV9 encoding FKRP gene) clinical trials

ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

This study is testing a new gene therapy, called ATA-100, for people with a rare muscle condition called LGMDR9. We want to see if it's safe and how it affects the body. Up to six adults will take part, and we'll watch them closely for five years.