Trials / Sponsors / Rare Trait Hope
Rare Trait Hope
Academic / Other · 1 registered clinical trial — 1 currently recruiting.
| Status | Trial | Phase | Started |
|---|---|---|---|
| Not Yet Recruiting | Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU) Aspartylglucosaminuria, Aspartylglucosamidase (AGA) Deficiency | Phase 1 / Phase 2 | 2026-05-01 |