Innovation In TreatmentSLDB's unique microdystrophin may have advantages over other designs that could improve impact on functional outcomes.
Regulatory SupportThe FDA has approved Solid Biosciences' IND application for SGT-212, a gene therapy for Friedreich's ataxia, showing regulatory support for the treatment.
Therapeutic PotentialSGT-212 has demonstrated significant preclinical success in improving neurological and cardiac functions in animal models, indicating its potential effectiveness.