2 clinical trials found.
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Sangamo Fabry disease gene therapy study (ST-920-201)
Fabry disease is caused by mutations in the GLA gene. The GLA gene encodes the lysosomal enzyme alpha-galactosidase A, or a-Gal A for short. When ...Phase
Interventional
TRIAL ROLE: Sub Investigator
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A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
The purpose of this study is to find out if an investigational new drug called ATB200 and AT2221 can help people with Pompe disease and ...Phase
Interventional
TRIAL ROLE: Sub Investigator
