The phase 2b allows a follow up dosing for a period, which would be good for the boys and their families.
All participants completing the Treatment Period of the study will be offered to the enter an open label extension study of ATL1102 conducted under a separate protocol and will be given the highest dose tested in the study or the highest dose considered generally safe and well tolerated in the study. This will serve to further increase the safety and efficacy database.
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