A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
2022-06-27
عنوان الدراسة
A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations
داعم الدراسة
Logic Bio Therapeutics
دواء الدراسة
AAV-hHA-hMUT
رقم بروتوكول الدراسة
LB001-001
حالة الدراسة
Ongoing
موقع الدراسة
King Abdulaziz Medical City NG (Riyadh)
مرحلة الدراسة
1
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