• |
  • |
  • |
  • |
Donate

Project

LSEP (PTC743-CNS-005-LSEP)

Center:
Fiscal Year:
2024
Contact Information:
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.
Keyword(s):
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
Funding Source:
COVID-19 Related Data:
N/A