-
2025-523087-20-00
5
Duchenne muscular dystrophy
-
2025-523157-34-00
5
Rett syndrome
-
2025-523738-21-00
4
Fragile X Syndrome
-
2025-523750-14-00
5
refractory hypotension in distributive shock
-
2025-523835-20-00
7
Dravet Syndrome
-
2025-524397-42-00
10
Participants with relapsed HRNB and participants
with HRNB refractory
-
2025-524487-37-00
6
Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MP
-
2025-525124-10-00
4
Duchenne Muscular Dystrophy
-
2025-522339-32-00
9
2026-06-30
Neoplasm malignant
-
2025-524140-35-00
4
2026-06-29
Neuroblastoma or ganglioneuroblastoma (nodular or intermixed)
-
2022-502910-10-00
7
2026-06-26
Propionic Acidemia
-
2025-524029-41-00
5
2026-06-22
Cardiomyopathy, tetralogy of Fallot, or complex congenital heart disea
-
2025-520993-20-00
4
2026-05-19
Generalized Myasthenia Gravis
-
2025-520988-41-00
4
2026-05-15
Immunoglobulin G4 related Disease (IgG4-RD)
-
2024-511593-70-00
5
2026-04-27
Polyarticular-course juvenile idiopathic arthritis
-
2024-519830-22-00
5
2026-04-27
Heart failure due to systemic left ventricular systolic dysfunction
-
2024-518615-19-00
5
2026-04-22
Major Depressive Disorder
-
2025-522191-86-00
5
2026-04-02
Focal segmental glomerulosclerosis
-
2023-503293-21-00
5
2026-03-25
Osteogenesis Imperfecta
-
2025-521858-42-00
5
2026-03-16
Hemophilia
-
2024-511508-18-00
9
2026-01-05
Chronic graft versus host disease.
-
2023-508547-51-00
5
2025-11-18
Aetiological treatment of moderate-to-severe allergic rhinitis/rhinoco
-
2025-520998-39-00
5
2025-11-07
Congenital pseudarthrosis of the tibia
-
2023-507192-22-00
7
2025-10-24
Methyl CpG binding protein 2 (MECP2) Duplication Syndrome (MDS)