-
2025-522949-22-00
5
Duchenne muscular dystrophy
-
2025-523087-20-00
5
Duchenne muscular dystrophy
-
2025-523096-34-00
5
B-cell Acute Lymphoblastic Leukemia
-
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
-
2025-524029-41-00
5
Cardiomyopathy, tetralogy of Fallot, or complex congenital heart disea
-
2025-524140-35-00
4
Neuroblastoma or ganglioneuroblastoma (nodular or intermixed)
-
2025-524264-38-00
6
Infectious diseases
-
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
-
2025-522170-36-00
5
2026-02-03
Diabetes Mellitus, Type 1
-
2025-521797-34-00
5
2026-01-20
Diabetes Mellitus, Type 1
-
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)
-
2024-514974-39-00
5
2025-10-10
Developmental and Epileptic Encephalopathies (DEEs)
-
2024-518645-17-00
5
2025-10-07
Blood and lymphatic diseases