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Results for “Aspartylglucosamidase (AGA) Deficiency”

2 trials

Showing 2 of 2 results

Not applicableEnded earlyNCT03853876
What this trial is testing

A Natural History Study of Aspartylglucosaminuria

Who this might be right for
AspartylglucosaminuriaAspartylglucosamidase (AGA) DeficiencyLysosomal Storage Diseases
Neurogene Inc. 8
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07530796
What this trial is testing

Safety and Efficacy of scAAV9/AGA Gene Therapy in Participants With Aspartylglucosaminuria (AGU)

Who this might be right for
AspartylglucosaminuriaAspartylglucosamidase (AGA) Deficiency
Rare Trait Hope 9

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