Functional gait training is a safe, feasible, and effective intervention to improve walking ability. Functional gait training shows larger positive effects on walking speed than standard physical therapy. Walking endurance and gait-related gross motor function can also benefit from functional gait training. Addition of virtual reality and biofeedback shows promise to increase engagement and improve outcomes.

Nusinersen (Spinraza®) improves survival of infants with hereditary proximal spinal muscular atrophy and motor function in children up to 12 years. Population-based assessments of treatment efficacy are limited and confined to select cohorts of patients. We performed a nationwide, population-based, single-center cohort study in children with spinal muscular atrophy younger than 9.5 years at start of treatment in line with reimbursement criteria in the Netherlands. We assessed age-relevant motor function scores, the need for tube feeding, hours of ventilatory support and documented adverse events. We used linear mixed modeling to assess treatment effects. We compared motor function during treatment with natural history data and to individual trajectories of muscle strength and motor function before the start of treatment. We included 71 out of 72 Dutch children who were treated (median age 54 months; range 0-117) and followed them for a median of 38 months (range 5-52). We observed improvement of motor function in 72% and stabilization in another 18% of the symptomatic children, which differed from the natural disease course in a matched cohort of which we had previously collected natural history data. Longitudinal analysis showed that motor function improved up to a median of 24 months (range 12-30) of treatment after which it stabilized. Shorter disease duration at start of treatment resulted in better treatment efficacy (p < 0.01). Sixteen children (23%) achieved new motor milestones. Bulbar and respiratory function did not improve significantly during treatment. In 15 patients from whom treatment-naïve data were available, the pre-treatment trajectory of motor function decline changed to stabilization or improvement after the start of treatment. We documented 82 adverse events after 934 injections (9%) in 45 patients. None of the adverse events led to treatment discontinuation. Intrathecal nusinersen treatment is safe and improves or stabilizes motor function in 90% of young children with spinal muscular atrophy types 1c–3a. We did not observe improvement of respiratory and bulbar functions.

People with cerebral palsy are often tired and may complain about fatigue. In this study we describe the development of fatigue over a mean period of 3 years. We found that fatigue is more or less stable over time on a group level. On an individual level there can be important changes, both increases and decreases in fatigue-related symptoms. We did not find any known risk factors for fatigue (for example body mass index, age) to be predictive of change in fatigue over time. We learned that adults with CP, who experience fatigue, are likely to remain fatigued in the future. We therefore think it is important for care providers to monitor and talk about fatigue with adults with CP. We would suggest care providers begin this discussion at a much younger age to influence fatigue later in life. Objectives: To describe the course of fatigue over a 3-year follow-up period in adults with cerebral palsy and to investigate the association of known determinants of fatigue (i.e. demographic characteristics and/or body composition) with change in fatigue. Methods: Forty-one adults with cerebral palsy from a previous study of fatigue were invited to participate in a follow-up study. Twenty-three adults with cerebral palsy (Gross Motor Function Classification System (GMFCS) levels I-V; mean age 38 years 2 months, standard deviation (SD) 14 years 1 month)) agreed to participate (convenience sample). Fatigue was measured with the Fatigue Impact and Severity Self-Assessment (FISSA, range 31-157) questionnaire. The course of fatigue is described at group, subgroup (GMFCS) and individual levels. Results: The mean FISSA score for all participants was 84.0 (SD 27.7) at baseline and 91.7 (SD 26.7) at follow-up. Despite variations among individuals in the change of fatigue, there was no statistically significant difference in FISSA score over time (p = 0.087, 95% confidence interval (95% CI)-16.7 to 1.22). No known determinants of fatigue predictive of change in FISSA scores were found. Discussion: Fatigue appears to be relatively stable within adults with cerebral palsy over time, with a variable presentation between individuals and across GMFCS levels. Care providers should monitor and discuss fatigue in young individuals with cerebral palsy in order to attenuate fatigue later in life.

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