Case Study

Duchenne Muscular Dystrophy Trial Case Study

Old Woman In Wheelchair

Administered in two phases, the pediatric study starts with patients aged 7 to 13 and goes up to young adults. During initial treatment the patients go through a phase of double-blind IMP administration before moving into an open label phase. Once that is completed the patients can move into a long-term extension study where patients can remain on treatment for up to 5 years.


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