Gene Transfer Study in Patients With Late Onset Pompe Disease Post author: Post published:March 1, 2026 Post category: Continue ReadingGene Transfer Study in Patients With Late Onset Pompe Disease
Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Type 1 Diabetes Post author: Post published:March 1, 2026 Post category: Continue ReadingPancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Type 1 Diabetes
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients Post author: Post published:March 1, 2026 Post category: Continue ReadingA Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis Post author: Post published:March 1, 2026 Post category: Continue ReadingStudy of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis Post author: Post published:March 1, 2026 Post category: Continue ReadingSafety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease Post author: Post published:March 1, 2026 Post category: Continue ReadingA Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR) Post author: Post published:March 1, 2026 Post category: Continue ReadingDose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
