-
2025-521304-21-00
Type 1 diabetes 2026-03-13
-
2025-522729-36-00
Chronic Hepatitis Delta. 2026-03-11
-
2024-519674-40-00
BAG3 Mutation Associated Dilated Cardiomyopathy 2026-02-05
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2022-501607-27-00
Relapsed or refractory B-cell Non-Hodgkin lymphoma 2026-01-06
-
2025-521672-60-00
Metastatic or locally advanced SST2-positive neuroendoc 2025-12-19
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2025-521299-76-00
Relapsed or refractory acute leukaemia or Myelodysplast 2025-12-18
-
2023-507192-22-00
Methyl CpG binding protein 2 (MECP2) Duplication Syndro 2025-10-24
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2025-522261-30-00
Parkinson disease 2025-10-01
-
2024-518144-20-00
Primary Hyperoxaluria Type 1 (PH1) 2025-09-03
-
2024-517500-11-00
Colorectal Cancer 2025-07-15
-
2023-510283-12-00
Sickle Cell Disease 2025-06-12
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2023-506842-22-00
Recurrent endometrial cancer, recurrent epithelial ovar 2025-05-19
-
2024-518368-10-00
Recessive dystrophic epidermolysis bullosa (RDEB) 2025-01-29
-
2023-507536-21-00
Advanced Solid Tumors 2024-10-04
-
2023-505605-16-00
Metastatic Clear Cell Renal Cell Carcinoma (ccRCC) or p 2024-04-24
-
2022-502285-24-00
Recurrent prostate cancer 2024-03-01
-
2022-500293-34-01
Bronchopulmonary Dysplasia 2023-12-28
-
2023-504506-11-00
Advanced Solid Tumors 2023-10-09
-
2022-501904-83-00
Ovarian Cancer 2023-09-11
-
2023-504254-35-00
Unresectable locally advanced or metastatic solid tumor 2023-06-20
-
2023-503795-24-00
Avanced Non-Small Cell Lung Cancer 2022-09-16
-
2022-502873-40-00
Wilson disease 2022-06-13
-
2023-509303-32-00
Pompe Disease (also known as glycogen storage disease t 2021-10-19
-
2023-510509-17-00
Relapsed and/or refractory Acute Myeloid Leukemia 2020-06-17