Clinical trials: Aspartylglucosaminuria, open to volunteers
Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)
The goal of this clinical trial is to learn if the treatment is a safe, tolerable, and efficacious treatment for adults and children with Aspartylglucosaminuria (AGU).
- Trial name
- NCT07530796
- Testing
- scAAV9/AGA
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Rare Trait Hope
Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency
AGU Registry and NHS
The Aspartylglucosaminuria (AGU) Registry and Natural History Study (AGU Registry) aims to gain a more comprehensive understanding of Aspartylglucosaminuria (AGU) disease through an in-depth understanding of the disease…
- Trial name
- NCT07903402
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Rare Trait Hope