-
2024-518645-17-00
5
2025-10-07
Blood and lymphatic diseases
-
2025-521055-23-00
10
2025-10-07
Chronic Idiopathic thrombocytopenic purpura
-
2022-501867-42-01
9
2025-09-08
Acute Lymphoblastic Leukemia, in Relapse
Lymphoblastic Lymphoma (Precu
-
2022-501866-22-00
9
2025-08-26
Acute Lymphoblastic Leukemia, in Relapse
Lymphoblastic Lymphoma (Precu
-
2023-509909-74-00
4
2025-08-13
Pruritus
-
2024-518973-32-00
5
2025-07-03
Chronic Graft-Versus-Host Disease
-
2024-514239-21-00
3
2025-05-21
Spinal Muscular Atrophy
-
2024-516617-20-00
5
2025-05-21
T-cell acute lymphoblastic Lymphoma
-
2023-505946-24-00
5
2025-05-15
Primary Immunodeficiency Diseases
-
2024-512600-19-00
5
2025-04-14
Angelman Syndrome
-
2022-501332-42-00
5
2025-02-26
Relapsing Remitting Multiple Sclerosis
-
2024-512991-36-00
5
2024-10-24
Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
-
2022-503112-17-00
5
2024-10-16
Limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4) or β-sarcoglyca
-
2024-512171-12-00
4
2024-09-27
PMM2-CDG
-
2022-502289-25-00
5
2024-08-28
Moderate to Severe Active Systemic Lupus Erythematosus
-
2023-504773-20-00
5
2024-08-01
Active Chronic Pouchitis
-
2022-502050-14-00
5
2024-06-04
Crohn's disease
-
2023-506026-36-00
5
2024-04-29
Juvenile Psoriatic Arthritis
-
2022-501599-25-00
5
2024-03-19
juvenile idiopathic arthritis
-
2023-503521-19-00
5
2024-02-09
Duchenne muscular dystrophy (DMD)
-
2022-501980-42-00
5
2023-12-21
Anemia associated with Chronic Kidney Disease
-
2022-501788-41-00
5
2023-11-14
Ulcerative Colitis
-
2023-508218-41-00
5
2023-08-17
Autosomal Recessive Polycystic Kidney Disease
-
2023-505140-19-00
5
2023-07-31
Childhood Idiopathic Nephrotic Syndrome