VISION- DMD’s primary ambition is to bring Valmorolone to clinic by 2020 as an innovative affordable therapy that delivers a significant improvement on the current therapeutic approach and standard of care for all DMD patients, with the potential of amplifying the efficacy of other DMD stratified therapies under development.
The Phase 2 clinical studies proposed by VISION-DMD will develop the evidence base for the most appropriate strategy of the Marketing Authorisation Application pathway, while also delivering innovations in pharmacodynamic biomarkers and outcome measures to the broader neuromuscular drug development community.
