Six minute walk test in type III spinal muscular atrophy: A 12month longitudinal study

Six-minute walk test is reliable and valid in spinal muscular atrophy

“…Because SMA is a slowly progressive disorder, sensitivity to change can only be examined in the context of a clinical intervention trial. Additional prospective studies are needed to confirm and extend the findings of this study with respect to further identifying measurement error, confirm responsiveness with previous studies, and confirm minimal important change (MIC). The MIC is needed to interpret the observed changes and determine the success of an intervention.…”

“…Responsiveness of the 6MWT in SMA was described as a mean change of ‐1.46 meters or a nominal change in percent‐predicted distance (51.4% to 50.6%), revealing that the 6MWT is stable over a 12‐month period, similar to other functional outcome measures in SMA . Furthermore, the magnitude of change was not related to age or baseline 6MWT distance . The distinctly low percent of predicted distances on the 6MWT indicate that a ceiling effect is unlikely to limit the responsiveness of the measure to a therapeutic intervention in SMA.…”

Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy

“…Similar to earlier work, [5][6][7][8] the ImagingDMD group found that boys >7 years old had significant declines in function over 1 year for both the 10-meter walk/ run test and the 6MWT. Although some studies have limited their enrollment of DMD subjects to those ≥7 years old, [9][10][11][12] and others have limited enrollment to ≥6 years old, [13][14][15] the baseline performance values on multiple clinical endpoints may be better predictors of the likelihood of functional decline over 1 year than a simple consideration of age.…”

Fatigability in spinal muscular atrophy: validity and reliability of endurance shuttle tests

“…The ESBBT is the first validated and sensitive fatigability test for proximal arm function in SMA and may be complementary to outcome measures that focus on arm motor function such as the Revised Upper Limb Measure (RULM), by adding the dimension of endurance [26]. Few studies have addressed the prevalence of fatigability and the variability in endurance capacity between ambulatory patients [24,27]. Our results show that most ambulatory patients do show fatigability during walking, but that the moment at which that occurs is highly variable.…”