-
2025-521581-10-00
Neoplasms, Prostate 2026-01-16
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2022-501607-27-00
Relapsed or refractory B-cell Non-Hodgkin lymphoma 2026-01-06
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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
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2025-520623-24-00
MTAP-deleted Solid Tumors 2025-11-19
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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
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2024-518144-20-00
Primary Hyperoxaluria Type 1 (PH1) 2025-09-03
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2022-502910-10-00
Propionic Acidemia 2025-08-11
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2024-517499-39-00
Duchenne Muscular Dystrophy 2025-07-16
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2024-517500-11-00
Colorectal Cancer 2025-07-15
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2024-512011-45-00
Unresectable locally advanced or metastatic colorectal 2025-06-17
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2024-515459-39-00
Advanced or Metastatic Squamous Solid Tumors 2025-05-08
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2024-518368-10-00
Recessive dystrophic epidermolysis bullosa (RDEB) 2025-01-29
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2023-507536-21-00
Advanced Solid Tumors 2024-10-04
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2023-505605-16-00
Metastatic Clear Cell Renal Cell Carcinoma (ccRCC) or p 2024-04-24
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2022-502285-24-00
Recurrent prostate cancer 2024-03-01
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2022-500293-34-01
Bronchopulmonary Dysplasia 2023-12-28
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2023-504254-35-00
Unresectable locally advanced or metastatic solid tumor 2023-06-20
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2023-503795-24-00
Avanced Non-Small Cell Lung Cancer 2022-09-16
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2022-502873-40-00
Wilson disease 2022-06-13
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2023-509303-32-00
Pompe Disease (also known as glycogen storage disease t 2021-10-19
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2023-510509-17-00
Relapsed and/or refractory Acute Myeloid Leukemia 2020-06-17
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2024-517762-42-01
recalcitrant lower limb nonunion 2018-08-02