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2025-522233-62-00
Advanced cancer; breast cancer; metastatic gastric / ga 2025-12-12
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2024-516602-28-00
Advanced Solid Tumors 2025-11-21
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2025-522719-40-00
CTNNB1 syndrome 2025-11-21
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2024-513332-17-00
KCNT1-related Developmental and Epileptic Encephalopath 2025-11-19
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2025-521227-70-00
Patients with locally advanced or metastatic prostate c 2025-11-13
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2024-511236-27-00
High grade serous or endometroid ovarian cancer 2025-11-12
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2024-517281-42-00
Advanced solid tumors 2025-11-12
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2025-521273-14-00
Solid neoplasms of the digestive system (primary and/or 2025-11-10
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2023-506384-34-00
Locally advanced or metastatic urothelial carcinoma 2025-11-05
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2022-500703-49-01
Duchenne muscular dystrophy 2025-11-05
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2023-507596-22-00
Neuroblastoma 2025-11-05
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2024-517765-16-00
Patients with Chronic Limb Threatening Ischemia (CLTI) 2025-11-01
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2024-517684-23-00
Cartilage defect of the knee joint 2025-10-23
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2024-514110-12-00
Thyroid Eye Disease 2025-10-22
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2024-512421-92-00
Unresectable, Locally Advanced or Metastatic Cancer 2025-10-14
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2024-512498-29-00
hearing loss 2025-09-24
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2022-503063-15-00
Multiple myeloma. 2025-09-22
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2024-519966-31-00
Relapsed/refractory Acute Lymphoblastic T Leukemia (T-A 2025-09-08
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2024-520395-99-00
Advanced solid tumors 2025-08-12
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2024-513900-32-00
Pancreatic Ductal Adenocarcinoma 2025-07-03
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2024-516019-26-00
Ductal adenocarcinoma of pancreas 2025-06-25
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2024-516900-41-00
Soft Tissue Sarcoma 2025-06-24
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2024-514501-57-00
Duchenne muscular dystrophy 2025-06-20
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2023-507260-40-00
Hemophilia B 2025-06-16