-
2024-515059-39-00
5
2024-02-09
Marfan Syndrome
Diseases related to Marfan syndrome (rare diseases wit
-
2022-502113-28-00
5
2024-02-01
Follicular lymphoma
-
2022-503092-28-00
5
2024-01-08
Relapsed/Refractory Follicular Lymphoma
-
2023-505911-19-00
4
2024-01-05
Acute Myeloid Leukemia
-
2023-504020-25-00
4
2023-12-28
Idiopathic Growth Hormone Deficiency
-
2022-502046-28-00
5
2023-12-22
HER2-Positive, PIK3CA Mutated, Locally Advanced or Metastatic Breast C
-
2022-501941-63-00
7
2023-12-13
Multiple myeloma
-
2024-517628-20-00
11
2023-10-24
hypertension
-
2023-503906-35-00
9
2023-10-11
Severe or Moderate Hemophilia A with or without factor VIII inhibitors
-
2022-501515-14-00
5
2023-09-29
Newly Diagnosed Multiple Myeloma (NDMM)
-
2024-514433-38-00
9
2023-09-28
fibrotic interstitial lung disease
-
2022-502281-25-00
5
2023-09-27
Focal Onset Seizures
-
2022-502732-39-00
10
2023-09-01
Previously treated non-ocular advanced melanoma
-
2024-518628-57-00
5
2023-08-01
Dravet syndrome
-
2024-517412-29-00
5
2023-07-10
arrhythmogenic in the old space of cardiomyopathy
-
2023-507171-22-00
9
2023-06-21
Untreated Advanced or Metastatic Non-Small Cell Lung Cancer
-
2024-513312-95-00
7
2023-05-30
Part B+C. Primary central nervous system lymphoma (PCNSL)
-
2023-504263-16-00
5
2023-05-25
This is an extension study designed to provide continued treatment for
-
2023-504861-22-00
4
2023-05-05
Pulmonary arterial hypertension
-
2023-510336-36-00
5
2023-05-05
Paroxysmal nocturnal hemoglobinuria (PNH)
-
2022-501238-52-00
10
2023-03-23
Heart failure due to systemic left ventricular systolic dysfunction
-
2022-501636-42-00
9
2023-03-21
Advanced RET-altered malignancies
-
2023-507396-21-00
4
2023-03-16
transfusion-dependent α- or β-thalassemia or low-risk myelodysplastic
-
2023-507278-41-00
5
2023-02-10
Familial hypercholsterolemia