Fig. 1.

Consensus was reached that category 1 drugs should be used as first-line treatment in DMD-naive patients unless the disease course is considered probably highly active (see A25). Efficacy category 2 and 3 DMDs should be used if category 1 drugs have failed to control the disease. However, apart from this well-known escalation approach, category 2 and 3 agents should also be offered to DMD-naive patients whose disease course is considered probably highly active from the beginning (see A29). The choice of category 1 to 3 thus follows both a step-in and a step-up approach. (OLU, off-label use)