Badanie nad bezpieczeństwem i skutecznością SPK-3006 u dorosłych z późno ujawniającą się chorobą Pompego

Pompe Disease, also known as glycogen storage disease type II, is a rare genetic disorder that affects the body's ability to break down glycogen, a type of sugar stored in muscles. This study focuses on adults with a form of this disease called late-onset Pompe Disease. The purpose of the study is to evaluate the safety and tolerability of a new treatment called SPK-3006. This treatment involves a single intravenous infusion, which means it is given directly into a vein.

The study will test different doses of SPK-3006 to find out how safe it is and how well it is tolerated by participants. The treatment contains a special substance called vanglusagene ensiparvovec, which is designed to help the body produce an important enzyme that is lacking in people with Pompe Disease. Participants will receive the treatment and be monitored for any side effects or changes in their health.

Throughout the study, researchers will observe how participants respond to the treatment, including any changes in their ability to walk and their lung function. The study will also look at how the body processes the treatment and any potential immune responses. This research aims to provide valuable information about the potential benefits and risks of SPK-3006 for people with late-onset Pompe Disease.