Project Description:
Title: SPIES-006: An intermediate size expanded access protocol of Elamipretide for subcutaneous injection in patients with genetically confirmed rare diseases with known mitochondrial dysfunction
Role: Site PI
Funding: Stealth BioTherapeutics
November 2019-present
Summary: This study is an open-label expanded access project treating patients with mitochondrial disease (MEGDEL, SANDO, NARP, MELAS).
Core Function(s):
Performing Research or Evaluation, Continuing Education/Community Training
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