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| Name | Class |
|---|---|
| PTC Therapeutics | INDUSTRY |
The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" with liquid Vatiquinone through an intermediate patient population expanded access protocol.
This expanded access protocol serves to make Vatiquinone accessible to subjects with inherited mitochondrial disease who previously participated in a Vatiquinone clinical trial at the Medical University of South Carolina (MUSC) who in the opinion of the investigator would benefit from continued access to liquid Vatiquinone.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Vatiquinone | Drug | Vatiquinone will be administered per dose and schedule specified in the arm description. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Thomas Koch, MD | Medical University of South Carolina | Principal Investigator |
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| ID | Term |
|---|---|
| D028361 | Mitochondrial Diseases |
| ID | Term |
|---|---|
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C571746 | alpha-tocotrienol quinone |
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