AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.
- Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.
- "Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease," said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i.
- "Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy.
- The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life."