SPACE.

Methods	Phase II, randomised, double‐blind, placebo‐controlled cross‐over trial
Participants	45 participants with SMA types II, III and IV, aged ≥ 12 years
Interventions	Oral pyridostigmine (days 1–3: 2 mg/kg/day over 4 doses per day; days 4–7: 4 mg/kg/day over 4 doses per day; weeks 2–8: 6 mg/kg/day over 5 doses per day) or oral placebo (days 1–3: 2 mg/kg/day over 4 doses per day; days 4–7: 4 mg/kg/day over 4 doses per day; weeks 2–8: 6 mg/kg/day over 5 doses per day). Cross‐over took place after 8 weeks with 1 week of washout.
Outcomes	Change in time to complete repeated 9 hole peg test, change in MFM scores, change in time to complete shuttle walk test, time to complete shuttle box and block test, change in pulmonary function (FVC), change in SMAFRS, change in PedsQL, fatigue and fatigability questionnaires, VAS scores, change in CMAP, and change in decremental response during repetitive nerve stimulation.
Notes	Study completed (January 2018), but no results are available yet.

4‐AP: 4‐aminopyridine; 6MWT: 6‐minute walk test; CMAP: compound muscle action potential; FVC: forced vital capacity; HFMS(E): Hammersmith Functional Motor Scale (Expanded); HHD: hand‐held dynamometry; MFM: Motor Function Measure; MEP: maximum expiratory pressure; MIP: maximum inspiratory pressure; PedsQL: Pediatric Quality of Life Inventory; RULM: Revised Upper Limb Module; TUG: Time Up and Go; RULM; Revised Upper Limb Module; SMA: spinal muscular atrophy; SMN: survival motor neuron; SMAFRS: Spinal Muscular Atrophy Functional Rating Scale; TUG: Timed Up and Go; VAS: visual analogue scale; WHO: World Health Organization.