Filling the gaps in knowledge translation: Physical therapy recommendations for individuals with spinal muscular atrophy compared to standard of care guidelines

Trenkle, Jessica; Brugman, Jessica; Peterson, Anne; Roback, Katherine; Krosschell, Kristin J.

doi:10.1016/j.nmd.2021.02.011

Cited by 7 publications

(6 citation statements)

References 38 publications

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“…5,6,9,18 In addition, consensus guidelines for children with Batten disease and SMA have recommended physical activity interventions, such as strengthening and stretching, to be performed at a minimum of 2 to 3 times per week. 9,10,12,44 Although this may not be considered "high frequency," it is a higher frequency than the child in this case report was previously receiving (1 time per week). 45 In addition, there are many studies supporting the efficacy of high-dose PT interventions (several times per week) for motor change for children with CP, a nonprogressive neurologic diagnosis.…”

Section: Discussionmentioning

confidence: 66%

“…High-dose PT interventions for other progressive and nonprogressive neurologic diagnoses are emerging and promising for improvements in motor skill development and quality of life 5,6,9,18. In addition, consensus guidelines for children with Batten disease and SMA have recommended physical activity interventions, such as strengthening and stretching, to be performed at a minimum of 2 to 3 times per week 9,10,12,44. Although this may not be considered “high frequency,” it is a higher frequency than the child in this case report was previously receiving (1 time per week) 45.…”

Section: Discussionmentioning

confidence: 99%

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Daily Outpatient Physical Therapy for a Toddler With a Neurodegenerative Disease: A Case Report

Bican

Ferrante

Hendershot

et al. 2022

Pediatric Physical Therapy

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Purpose: This case report highlights the potential value of delivering a high-dose physical therapy (PT) intervention for a child with a neurodegenerative disease. We include developmental outcomes for a 23-month-old toddler with biallelic TBCD gene mutations following daily outpatient PT. Summary of Key Points: The child had clinical improvements in gross and fine motor, cognition, expressive and receptive language, socioemotional, and adaptive behavior function as determined through Goal Attainment Scaling, Gross Motor Function Measure, and Bayley Scales of Infant and Toddler Development following daily PT intervention. Statement of Conclusion and Recommendations for Clinical Practice: High-dose outpatient PT may be beneficial for a child with a neurodegenerative disease at some time frames. In selected cases, if the neurodegenerative disease slowly progresses, high-dose PT may be a treatment option to promote motor change.

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Section: Discussionmentioning

confidence: 66%

Section: Discussionmentioning

confidence: 99%

Daily Outpatient Physical Therapy for a Toddler With a Neurodegenerative Disease: A Case Report

Bican

Ferrante

Hendershot

et al. 2022

Pediatric Physical Therapy

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“…These findings affirm the safety and tolerance of interventions in this population. 2,23,46 Previous studies indicate that exercise, including arm ergometer training, resisted range of motion exercises, and strength training, improves muscle strength in children with DMD and BMD. 22,30,47,48 The present study, consistent with literature, showed that VR and biofeedback training improved muscle strength, whereas continuing a regular rehabilitation program preserved strength in this group.…”

Section: Discussionmentioning

confidence: 99%

Feasibility of virtual reality and comparison of its effectiveness to biofeedback in children with Duchenne and Becker muscular dystrophies

Kurt‐Aydin,

Savaş‐Kalender,

Tarsuslu

et al. 2024

Muscle and Nerve

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Introduction/AimsThe utilization of virtual reality (VR) and biofeedback training, while effective in diverse populations, remains limited in the treatment of Duchenne and Becker muscular dystrophies (D/BMD). This study aimed to determine the feasibility of VR in children with D/BMD and compare the effectiveness of VR and biofeedback in children with D/BMD.MethodsThe study included 25 children with D/BMD. Eight children in the control group participated in a routine follow‐up rehabilitation program, while the remaining children were randomly assigned to the VR (n = 9) and biofeedback (n = 8) groups for a 12‐week intervention. The following evaluations were performed before, during (week 6), and after treatment: Muscle pain and cramps, laboratory studies, muscle strength, timed performance, function (Motor Function Measurement Scale‐32, Vignos, and Brooke Scales), and balance (Pediatric Functional Reach Test and Balance Master System). Motivation for rehabilitation was determined.ResultsThe median ages were 9.00 (VR), 8.75 (biofeedback), and 7.00 (control) years. The study found no significant differences between groups in pretreatment assessments for most measures, except for tandem step width (p < .05). VR and biofeedback interventions significantly improved various aspects (pain intensity, cramp frequency, cramp severity, muscle strength, timed performance, functional level, and balance) in children with D/BMD (p < .05), while the conventional rehabilitation program maintained patients' current status without any changes. The study found VR and biofeedback equally effective, with VR maintaining children's motivation for rehabilitation longer (p < .05).DiscussionThe study showed that both VR and biofeedback appear to be effective for rehabilitation this population, but additional, larger studies are needed.

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“…Physical therapy is essential in managing muscle weakness, contractures, and maintaining range of motion (84,85). People affected by spinal muscular atrophy may experience limitations in their ability to fully articulate their joints due to muscle weakness.…”

Section: Physical Therapymentioning

confidence: 99%

Diving into progress: a review on current therapeutic advancements in spinal muscular atrophy

Bagga,

Singh,

Ram

et al. 2024

Front. Neurol.

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Spinal muscular atrophy (SMA) is an uncommon disorder associated with genes characterized by the gradual weakening and deterioration of muscles, often leading to substantial disability and premature mortality. Over the past decade, remarkable strides have been made in the field of SMA therapeutics, revolutionizing the landscape of patient care. One pivotal advancement is the development of gene-targeted therapies, such as nusinersen, onasemnogene abeparvovec and risdiplam which have demonstrated unprecedented efficacy in slowing disease progression. These therapies aim to address the root cause of SMA by targeting the survival motor neuron (SMN) gene, effectively restoring deficient SMN protein levels. The advent of these innovative approaches has transformed the prognosis for many SMA patients, offering a glimmer of hope where there was once limited therapeutic recourse. Furthermore, the emergence of small molecule compounds and RNA-targeting strategies has expanded the therapeutic arsenal against SMA. These novel interventions exhibit diverse mechanisms of action, including SMN protein stabilization and modulation of RNA splicing, showcasing the multifaceted nature of SMA treatment research. Collective efforts of pharmaceutical industries, research centers, and patient advocacy groups have played an important role in expediting the translation of scientific discoveries into visible clinical benefits. This review not only highlights the remarkable progress achieved in SMA therapeutics but also generates the ray of hope for the ongoing efforts required to enhance accessibility, optimize treatment strategies, rehabilitation (care and therapies) and ultimately pave the way for an improved quality of life for individuals affected by SMA.

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Filling the gaps in knowledge translation: Physical therapy recommendations for individuals with spinal muscular atrophy compared to standard of care guidelines

Cited by 7 publications

References 38 publications

Daily Outpatient Physical Therapy for a Toddler With a Neurodegenerative Disease: A Case Report

Daily Outpatient Physical Therapy for a Toddler With a Neurodegenerative Disease: A Case Report

Feasibility of virtual reality and comparison of its effectiveness to biofeedback in children with Duchenne and Becker muscular dystrophies

Diving into progress: a review on current therapeutic advancements in spinal muscular atrophy

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