A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease
Latest Information Update: 05 May 2024
At a glance
- Drugs GC-301 (Primary)
- Indications Glycogen storage disease type II
- Focus Adverse reactions; Therapeutic Use
- Sponsors GeneCradle Therapeutics
Most Recent Events
- 05 May 2024 New trial record