OBJECTIVES: Duchenne muscular dystrophy (DMD) is a rare recessive X-linked disorder caused by a defective gene coding for dystrophin, afflicting primarily boys. Symptom onset is in early childhood (3-5 years old). DMD is characterized by progressive symmetrical muscular weakness affecting proximal muscles more than distal muscles. Loss of independent ambulation occurs between the ages of 6 and 13 years. Cardiomyopathy and respiratory failure are the cause of death in young adulthood. The objective of this research was to review the role of clinical outcome assessments (COAs) in drug approval and labeling of products authorized for the treatment of DMD in Europe and in the USA. METHODS: This research was conducted through a systematic manual review of DMD specific EMA and FDA regulatory guidelines, product labeling and corresponding assessment reports. The PROLabels database was used for labeling claim identification. RESULTS: In its DMD-specific guideline (December 2015), EMA recommended the use of several COAs to explore motor function [e.g., 6-minute walk test (6WMT)], muscle strength, activities of daily living (e.g., Functional Independence Measure or Barthel Index), quality of life (e.g., PedsQL core and neuromuscular module), respiratory function, and cognitive impairment. Caregiver evaluation was also recommended. In its draft guidance (September 2015), FDA follows a similar approach, but does not suggest names of HRQL scales. Only one medicinal product, approved by the EMA, was identified, i.e., ataluren. The main COA labeling claim refers to improvement in distance assessed by the 6MWT. HRQL was evaluated using the PedsQL but not included in the label, since only positive trends favouring ataluren vs placebo in the physical function domain were measured. CONCLUSIONS: With only one product approved for DMD treatment and no patient’s perspective included in its label, the choice of medication is awfully limited for patients (and their parents), who should deserve more research efforts.