T.O.4 Safety and efficacy results from a randomized, double-blind, placebo-controlled study of alglucosidase alfa for the treatment of Pompe disease in juveniles and adults

The 6‐minute walk test in Duchenne/Becker muscular dystrophy: Longitudinal observations

“…This level of decrement exceeds the changes in 6MWD of ∽10–15% that have been shown to be consistent with improving or worsening patient‐reported well‐being 32. This decrement also exceeds the mean treatment effects of 28–44 meters (8–13%) achieved in registration‐directed, randomized studies that employed change in 6MWD as the basis for approval of drugs for several different indications 14–17. Collectively, the data suggest that improving ambulatory capacity in boys with DBMD should continue to be a major treatment objective, but minimizing loss of ambulation would also remain a laudable therapeutic goal.…”

“…The 6‐minute walk test (6MWT) was originally developed as an integrated assessment of cardiac, respiratory, circulatory, and muscular capacity 12. More recently, the 6MWT has been used to evaluate functional capacity in neuromuscular diseases13, 14 and has served as the basis for regulatory registration of several drugs 14–17. In a short‐term study, we previously reported that a modified 6MWT is feasible, safe, and reliable in boys with DBMD who have not yet transitioned to fulltime wheelchair use 18.…”

“…We elected to contact the same boys with DBMD and healthy boys who had participated in our short‐term study to determine whether they would be willing to undergo repeat evaluation ∽1 year later. We selected this follow‐up period because it extended past the 6–12‐month treatment durations for trials that evaluated corticosteroids in DBMD,10 was similar to the period of evaluation in the ataluren registration study in DBMD,20 was consistent with durations of observation ranging from 6 to 18 months in registration trials that evaluated change in walking distance in other genetic disorders,14, 16, 17, 21 and acknowledged the challenge and expense of maintaining a longitudinal drug intervention project for ≥1 year.…”

Effects of balance strategy training in myasthenia gravis: A case study series

“…The 6‐minute walk test (6MWT) was administered according to a standardized protocol . The 6MWT was chosen because it has shown good test–retest reliability and is validated as a useful indicator of functional capacity and endurance in numerous populations, including patients with neuromuscular diseases . Several studies have also used this test to assess clinical changes after therapy …”

The 6‐minute walk test as a new outcome measure in Duchenne muscular dystrophy

“…More recently, its relevance to neuromuscular diseases has been established. In registration‐directed studies of laronidase in mucopolysaccharidosis type I (MPS I),17 idursulfase in mucopolysaccharidosis type II (MPS II),18 and alglucosidase‐α in Pompe disease,19 the 6MWT served as a primary outcome measure. Within the family of muscular dystrophies, the 6MWT has proved reliable and feasible in myotonic dystrophy type 1 (DM1) 20.…”