Phase |/||

Therapeutic Approach

Restoring or Replacing Dystrophin

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.


On October 1, 2020, Solid Biosciences Inc. shared that the FDA has lifted the Clinical Hold on the IGNITE DMD Clinical Trial.


This program is sponsored by Solid Biosciences.

Related Studies

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Media Library

JUNE 2019

Solid BioSciences Presents at the PPMD 2019 Annual Conference

JUNE 2018

Solid BioSciences Presents at the PPMD 2018 Annual Conference

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