Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy

Figures

• 

  Figure 1. Six-minute walk test distance for all patients at baseline (day 1 visit). 6MWT = 6-minute walk test.

• 

  Figure 2. Rapid decline and loss of ambulation in the eteplirsen-treated twin patients. 6MWT = 6-minute walk test.

• 

  Figure 3. Measures of pulmonary and cardiac function in patients A and B versus study mean through 240 weeks of eteplirsen treatment: (A) FVC%p and (B) LVEF. FVC%p = percent predicted forced vital capacity, LVEF = left ventricular ejection fraction.

• 

  Figure 4. Measures of upper limb function in patients A and B versus study mean through 240 weeks of eteplirsen treatment: (A) 9-Hole Peg Test; (B) grip strength; (C) elbow flexion; and (D) elbow extension. All data represent the right hand, which was the dominant hand for all 12 patients.

• 

  Figure 5. Dystrophin expression following 180 weeks of eteplirsen treatment, based on Western blot analysis and immunohistochemistry (A), Western blot image (B), PDPF (C), and the intensity of dystrophin fluorescent signal in muscle fibers (D). In panels A, C, and D, standard deviations are represented by error bars. In panel B, lanes in the Western blot image are identified left to right as the standard curve (4%, 2%, 1%, 0.5%, 0.25% of normal), patient samples (treated or untreated in blinded random order in next two lanes), and negative pooled DMD control sample. Patient samples representing patient A and patient B are identified in both figures. Note that either an unrelated patient sample representing an untreated (left figure) or treated (right figure) patient was run on the adjacent lane due to the random blinded nature that samples were evaluated. DMD = Duchenne muscular dystrophy, PDPF = percent dystrophin-positive fibers, tx = treated, untx = untreated.