title: A Randomized, Controlled Clinical Trial of Exercise in Patients with Spinal Muscular Atrophy: Methods and Baseline Characteristics.
abstract: BACKGROUND
  Spinal Muscular Atrophy (SMA) is a recessively-inherited neuromuscular disease characterized by weakness and muscle atrophy. Although anecdotal benefits from exercise have been noted, and despite promising pre-clinical and pilot reports, the effect of exercise has not been addressed in a controlled trial in SMA.
  
  
  OBJECTIVE
  To assess the effects of exercise on measures of function, strength, and exercise capacity in ambulatory SMA patients.
  
  
  METHODS
  An evaluator-blinded, randomized, controlled trial of aerobic and strengthening exercise in 14 ambulatory SMA patients aged 8-50 years. Patients will be randomized to either the exercise or control arm after the 1 month lead in period. During the first 6-months, the exercise group will receive the intervention while the other group serves as a control. After those 6 months, both groups will receive the intervention. The last 6-months of the study are designed to mimic real-world conditions where all participants are encouraged to continue on their own. Participants will be monitored throughout this 19 month study and will have in-person visits every three months. The primary outcome measure is the change in the total distance walked over 6-months on the six minute walk test (6MWT). Secondary outcome measures include maximal oxygen uptake (VO2 max), functional and strength assessments, pulmonary function, fatigue, and quality of life.
  
  
  CONCLUSIONS
  The result of this prospective, single blinded, randomized and controlled clinical trial of exercise on an established functional outcome measure will have impact on clinical practice by providing important guidance to clinical management of SMA patients.
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Montes
authors.affiliation: Biobehavioral Sciences Department, Teachers College, Columbia University, NY, USA.
authors.forename: Carol Ewing
authors.initials: CE
authors.lastname: Garber
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Samantha S
authors.initials: SS
authors.lastname: Kramer
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Megan J
authors.initials: MJ
authors.lastname: Montgomery
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Sally
authors.initials: S
authors.lastname: Dunaway
authors.affiliation: Biobehavioral Sciences Department, Teachers College, Columbia University, NY, USA.
authors.forename: Shirit
authors.initials: S
authors.lastname: Kamil-Rosenberg
authors.affiliation: Biobehavioral Sciences Department, Teachers College, Columbia University, NY, USA.
authors.forename: Brendan
authors.initials: B
authors.lastname: Carr
authors.affiliation: Department of Rehabilitation and Regenerative Medicine, Columbia University Medical Center, NY, USA.
authors.forename: Nancy E
authors.initials: NE
authors.lastname: Strauss
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Douglas
authors.initials: D
authors.lastname: Sproule
authors.affiliation: Department of Neurology, Columbia University Medical Center, NY, USA.
authors.forename: Darryl C
authors.initials: DC
authors.lastname: De Vivo