GNT0006/ATA-100 for treating limb girdle muscular dystrophy type R9 (FKRP-related)

GNT0006/ATA-100 is being developed to treat a rare muscle condition called limb-girdle muscular dystrophy type R9 (FKRP-related). This condition is caused by a change in a gene that helps muscles stay strong and work properly. When this gene does not function as it should, the muscles around the hips and shoulders gradually become weaker and smaller. Over time, people may find it harder to move and may eventually need a wheelchair. The condition can also affect the heart and breathing muscles, which may become life-threatening. There are currently no approved medicines that can stop or slow down the disease, and available care focuses on supportive care for managing symptoms and maintaining quality of life.