LSEP (PTC743-CNS-005-LSEP)
Project Description:
Title: LSEP (PTC743-CNS-005-LSEP): An open-label, safety study for previously treated Vatiquinone (PTC743) subjects with inherited mitochondrial disease
Role: Site PI
Funding: PTC Therapeutics
2022-present
Summary: This is an open-label expanded access program to combine subjects from previous studies receiving Vatiquinone to allow for continued treatment.
Core Function(s):
Continuing Education/Community Training
Area of Emphasis
Education & Early Intervention, Quality of Life
Target Audience:
Children/Adolescents with Disabilities/SHCN
Unserved or Under-served Populations:
Other
Primary Target Audience Geographic Descriptor:
National
COVID-19 Related Data:
N/A