Dynacure have now updated the Frequently Asked Questions document (FAQ) posted on their website and have included translated versions as well. You can access the new document here from the company’s own website: https://www.dynacure.com/patients-and-families/
Here are the main items to call your attention, following our patient community questions to Dynacure during our recent Myotubular Trust online Scientific Update. They have informed us that:-
- They expect initial data from their current Phase 1/2 adult study, ‘Unite-CNM’, in the second half of 2022 at the earliest. They are unable to give updates, timing or plans for the pediatric (child patient) study, until they first evaluate the initial adult data.
- They do not plan to enroll adult patients in the U.S. into ‘Unite-CNM’, for reasons that are explained in the FAQ.
- In terms of what happened with the Audentes/Astellas study and how that might impact Dynacure’s work they state that while their therapeutic approach is different, they have still taken the steps to increase the monitoring of liver parameters of patients after being dosed. In addition, they are progressing dose increases very slowly and have added experts on liver function to their panel of scientific advisors. Again, this is addressed more specifically in the updated FAQ document.
