-
2024-519494-19-00
5
2025-12-22
Nutritional and metabolic diseases
-
2024-520130-29-00
4
2025-12-17
Spinal Muscular Atrophy
-
2024-518465-10-00
4
2025-12-12
Multiple Autoimmune Glomerular Diseases
-
2024-519960-42-00
4
2025-12-11
PIK3CA-Related Overgrowth Spectrum (PROS)
-
2025-520896-13-00
5
2025-12-04
Friedreich’s ataxia
-
2025-521381-10-00
5
2025-12-03
Moderately to Severely Active Ulcerative Colitis
-
2025-521697-34-00
5
2025-12-01
Peanut allergy
-
2024-518895-30-00
4
2025-11-21
PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA
-
2024-515564-32-00
4
2025-11-20
Catecholaminergic Polymorphic Ventricular Tachycardia
-
2024-513332-17-00
7
2025-11-19
KCNT1-related Developmental and Epileptic Encephalopathy
-
2024-518683-13-00
6
2025-11-12
Acute lower Respiratory Tract Infection or acute rhinosinusitis
-
2024-518156-24-00
5
2025-11-10
Atopic Dermatitis
-
2024-511989-36-00
5
2025-09-24
Ewing Sarcoma
-
2024-517633-40-00
5
2025-09-18
Hidradenitis Suppurativa
-
2025-521196-32-00
5
2025-09-12
Venous malformation or low-flow venous/lymphatic malformation
-
2024-513171-41-00
5
2025-09-11
Vitiligo
-
2024-516412-17-00
5
2025-08-26
Developmental and Epileptic Encephalopathies (DEE)
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2023-508050-26-00
3
2025-07-17
AML
-
2024-518378-14-00
5
2025-07-11
Leber congenital amaurosis (LCA)
-
2024-518771-66-00
9
2025-07-01
Hepatoblastoma, Rhabdomyosarcoma
-
2024-520167-13-00
5
2025-06-17
Immunoglobulin A Nephropathy
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2023-504246-64-02
9
2025-06-04
Relapsed/refractory neuroblastoma and other solid tumours
-
2024-514937-39-00
5
2025-05-20
Dravet Syndrome
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2024-517584-23-00
9
2025-05-16
Duchenne Muscular Dystrophy