SGT-001 is an adeno-associated virus (AAV) mediated gene transfer that contains an engineered version of the dystrophin gene (microdystrophin). The microdystrophin gene in SGT-001 has been designed to produce a functional form of dystrophin protein in skeletal and cardiac muscles.
This study is currently on clinical hold.
On March 30, 2020, Solid Biosciences Inc. shared an update with the community regarding the IGNITE DMD clinical hold and COVID-19.
This program is sponsored by Solid Biosciences.
|Microdystrophin Gene Transfer Study in Adolescents and Children With DMD|
Solid BioSciences Presents at the PPMD 2019 Annual Conference
Solid BioSciences Presents at the PPMD 2018 Annual Conference