Selective voluntary motor control influences knee joint torque, work and power in children with spastic cerebral palsy.

Background: Children with spastic cerebral palsy (CP) have damage to the corticospinal tracts that are responsible for selective motor control (SMC). Force, velocity and timing of joint movement are related biomechanical features controlled by the corticospinal tracts (CSTs) that are important for skilled movement.

Research Question: Does SMC influence knee joint biomechanics in spastic CP?

Methods: In this prospective study, relationships between SMC and knee biomechanics (peak torque, total work, average power) across a range of velocities (0-300 deg/s) were assessed using an isokinetic dynamometer in 23 children with spastic CP.

Kinematic changes in gait in boys with Duchenne Muscular Dystrophy: Utility of the Gait Deviation Index, the Gait Profile Score and the Gait Variable Scores.

Background: Duchenne Muscular Dystrophy (DMD) is an X-linked muscle disorder caused by a mutation or deletion in the dystrophin gene. In boys with DMD, muscle weakness progresses in a proximal to distal pattern, leading to gait abnormalities at all joints, in all planes of motion. Longitudinal studies are imperative to quantify changes in gait function due to DMD and are of particular importance when examining the efficacy of treatment interventions.

A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy.

Background: Duchenne muscular dystrophy (DMD) is an X-linked recessive genetic disorder, that is characterized by progressive muscle degeneration and loss of ambulation between 7-13 years of age. Novel pharmacological agents targeting the genetic defects and disease mechanisms are becoming available; however, corticosteroid (CS) therapy remains the standard of care.

Objective: The purpose of this longitudinal study was to elucidate the effect of CS therapy on the rate of muscle strength and gross motor skill decline in boys with DMD and assess the sensitivity of selected outcome measures.

Longitudinal changes in energy cost during walking in boys with Duchenne Muscular Dystrophy (DMD).

Background: In boys with DMD, muscle weakness progresses in a proximal to distal pattern, leading to compensatory gait strategies, including hyperlordosis and equinus, that increase energy cost and accelerate the loss of walking capacity.

Research Question: The purpose of this study was to determine the changes in the energy cost of walking that occur with disease progression and to determine the optimal normalization scheme for the longitudinal assessment of the energy cost of walking in boys with DMD.

Methods: Energy cost was assessed with the COSMED K4b.

Gait outcomes following proximal tibial tumor resection and endoprosthetic reconstruction.

Background: Despite the proximal tibia being a common site of primary malignant bone tumors, there is limited information about gait function following proximal tibial tumor resection and endoprosthetic reconstruction (PTR).

Research Question: What is the impact of PTR on gait and quality of life?

Methods: This was a cross-sectional study of patients ≥18 years old who were ≥2 years post-PTR compared to a control group of similar age and sex distribution. Eighteen participants (9 PTR, 9 Control) were recruited.

Women with cerebral palsy: A qualitative study about their experiences with sexual and reproductive health education and services.

Purpose: To explore the recalled experiences of women with CP regarding sexual health education and services they received.

Methods: Semi-structured interviews and focus groups were conducted at four academic tertiary hospitals with 33 adult women with CP. Templates were used to ask about four key content domains: appointment planning (including planning for a gynecologic exam), accessibility of services, experiences with providers, and recommendations for improvement.

Brain Metabolism During A Lower Extremity Voluntary Movement Task in Children With Spastic Cerebral Palsy.

Reduced selective voluntary motor control (SVMC) is a primary impairment due to corticospinal tract (CST) injury in spastic cerebral palsy (CP). There are few studies of brain metabolism in CP and none have examined brain metabolism during a motor task. Nine children with bilateral spastic CP [Age: 6-11 years, Gross Motor Function Classification System (GMFCS) Levels II-V] completed this study.

MRI safety and imaging artifacts evaluated for a cannulated screw used for guided growth surgery.

Objective: Percutaneously-placed cannulated screws are the implant of choice for treatment of skeletal deformity associated with growing children that have spastic cerebral palsy (CP). These patients often require MRI examinations throughout their childhood to evaluate associated comorbidities and frequently for research protocols. There are concerns related to the use of MRI when metallic implants are present.

A common data language for clinical research studies: the National Institute of Neurological Disorders and Stroke and American Academy for Cerebral Palsy and Developmental Medicine Cerebral Palsy Common Data Elements Version 1.0 recommendations.

Unlabelled: To increase the efficiency and effectiveness of clinical research studies, cerebral palsy (CP) specific Common Data Elements (CDEs) were developed through a partnership between the National Institute of Neurological Disorders and Stroke (NINDS) and the American Academy of Cerebral Palsy and Developmental Medicine (AACPDM). International experts reviewed existing NINDS CDEs and tools used in studies of children and young people with CP. CDEs were compiled, subjected to internal review, and posted online for external public comment in September 2016.

Characterization of Strength and Function in Ambulatory Adults With GNE Myopathy.

Objective: To characterize the pattern and extent of muscle weakness and impact on physical functioning in adults with GNEM.

Methods: Strength and function were assessed in GNEM subjects (n = 47) using hand-held dynamometry, manual muscle testing, upper and lower extremity functional capacity tests, and the GNEM-Functional Activity Scale (GNEM-FAS).

Results: Profound upper and lower muscle weakness was measured using hand-held dynamometry in a characteristic pattern, previously described.

Longitudinal community walking activity in Duchenne muscular dystrophy.

Introduction: Natural history studies for Duchenne muscular dystrophy (DMD) have not included measures of community ambulation.

Methods: Step activity (SA) monitors quantified community ambulation in 42 boys (ages 4-16 years) with DMD with serial enrollment up to 5 years by using a repeated-measures mixed model. Additionally, data were compared with 10-meter walk/run (10mWR) speed to determine validity and sensitivity.

Stepping Up to Rethink the Future of Rehabilitation: IV STEP Considerations and Inspirations.

Background And Purpose: The IV STEP conference challenged presenters and participants to consider the state of science in rehabilitation, highlighting key area of progress since the previous STEP conference related to prediction, prevention, plasticity, and participation in rehabilitation.

Key Points: Emerging from the thought-provoking discussions was recognition of the progress we have made as a profession and a call for future growth. In this summary article, we present a recap of the key points and call for action.

Stepping Up to Rethink the Future of Rehabilitation: IV STEP Considerations and Inspirations.

Background And Purpose: The IV STEP conference challenged presenters and participants to consider the state of science in rehabilitation, highlighting key area of progress since the previous STEP conference related to prediction, prevention, plasticity, and participation in rehabilitation.

Key Points: Emerging from the thought-provoking discussions was recognition of the progress we have made as a profession and a call for future growth. In this summary article, we present a recap of the key points and call for action.

Reproductive healthcare experiences of women with cerebral palsy.

Background: Little is known about pregnancy rates in women with disabilities in general and even less is known about women with child-onset disabilities such as cerebral palsy (CP).

Hypothesis: We hypothesized that discussions about pregnancy with healthcare providers and pregnancy rates for woman with CP would be related to their functional levels.

Methods: Survey methodology was used to gather information about demographics, function, whether women were asked about their desire for children, pregnancy outcomes, and services offered during pregnancy and postpartum.

Safety, Tolerability, and Sensorimotor Effects of Extended-release Dalfampridine in Adults With Cerebral Palsy: A Pilot Study.

Purpose: The goal of this study was to evaluate the safety and tolerability of dalfampridine extended release (D-ER) in a pilot study of adults with cerebral palsy (CP) and limited ambulatory ability, and to explore drug effects on sensorimotor function.

Methods: An initial double-blind, single-dose crossover study was performed in 11 individuals randomized 1:1 to receive D-ER (10 mg) or placebo, followed by a 2-day washout period and the opposite treatment, with evaluation for safety and tolerability. A twice daily dosing, double-blind, placebo-controlled, crossover study was then performed.

Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and Perceived Quality of Life?

The aim of this study was to determine whether prednisone and deflazacort play a different role in child behavior and perceived health related psychosocial quality of life in ambulant boys with Duchenne Muscular Dystrophy. As part of a prospective natural-history study, parents of sixty-seven ambulant boys with DMD (27 taking prednisone, 15 taking deflazacort, 25 were steroid naïve) completed the Child Behavior Checklist (CBCL) for assessment of behavioral, emotional and social problems and both parents and boys with DMD completed the PedsQL™4.0 generic core scale short form.

Can Quantitative Muscle Strength and Functional Motor Ability Differentiate the Influence of Age and Corticosteroids in Ambulatory Boys with Duchenne Muscular Dystrophy?

Background: In the absence of a curative treatment for Duchenne Muscular Dystrophy (DMD), corticosteroid therapy (prednisone, deflazacort) has been adopted as the standard of care, as it slows the progression of muscle weakness and enables longer retention of functional mobility. The ongoing development of novel pharmacological agents that target the genetic defect underlying DMD offer hope for a significant alteration in disease progression; however, substantiation of therapeutic efficacy has proved challenging. Identifying functional outcomes sensitive to the early, subtle changes in muscle function has confounded clinical trials.

Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD) is an X-linked genetic neuromuscular disorder characterized by progressive proximal to distal muscle weakness. The success of randomized clinical trials for novel therapeutics depends on outcome measurements that are sensitive to change. As the development of motor skills may lead to functional improvements in young boys with DMD, their inclusion may potentially confound clinical trials.

Active Videogaming for Individuals with Severe Movement Disorders: Results from a Community Study.

Objective: Active videogaming (AVG) has potential to provide positive health outcomes for individuals with cerebral palsy (CP), but their use for individuals with severe motor impairments is limited. Our objective was to evaluate the accessibility and enjoyment of videogames using the Kinect™ (Microsoft, Redmond, WA) with the Flexible Action and Articulated Skeleton Toolkit (FAAST) system (University of Southern California Institute for Creative Technologies, Los Angeles, CA) for individuals with severely limiting CP.

Materials And Methods: A videogaming system was installed in a community center serving adults with CP, and a staff member was instructed in its use.

Bone density in premenopausal women and men under 50 years of age with cerebral palsy.

Objective: To determine bone mineral density (BMD) z scores in adults with cerebral palsy (CP), an understudied population.

Design: Cross-sectional.

Setting: Medical facility.

Health-related physical fitness for children with cerebral palsy.

Low levels of physical activity are a global health concern for all children. Children with cerebral palsy have even lower physical activity levels than their typically developing peers. Low levels of physical activity, and thus an increased risk for related chronic diseases, are associated with deficits in health-related physical fitness.

What are the functional outcomes of endoprosthestic reconstructions after tumor resection?

Background: The majority of published functional outcome data for tumor megaprostheses comes in the form of subjective functional outcome scores. Sparse objective data exist demonstrating functional results, activity levels, and efficiency of gait after endoprosthetic reconstruction in patients treated for orthopaedic tumors. Patients embarking on massive surgical operations, often in the setting of debilitating medical therapies, face mortality and a myriad of unknowns.