Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more than women, most commonly after age 50. Clinical features include weakness of the... more
Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more than women, most commonly after age 50. Clinical features include weakness of the quadriceps, finger flexors, ankle dorsiflexors, and dysphagia. The distribution of weakness is similar to Becker muscular dystrophy, where we previously reported improvement following intramuscular injection of an isoform of follistatin (FS344) by AAV1. For this clinical trial, rAAV1.CMV.huFS344, 6 × 10(11) vg/kg, was delivered to the quadriceps muscles of both legs of six sporadic inclusion body myositis subjects. The primary outcome for this trial was distance traveled for the 6-min walk test. The protocol included an exercise regimen for each participant. Performance, annualized to a median 1-year change, improved +56.0 m/year for treated subjects compared to a decline of -25.8 m/year (p = 0.01) in untreated subjects (n = 8), matched for age, gen...
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Technology, Skeletal muscle biology, Biomarkers, Treatment Outcome, Biopsy, and 15 moreBiological Sciences, Humans, Mice, Animals, Male, Follow-up studies, Gene Dosage, Aged, Middle Aged, Follistatin, Molecular Targeted Therapy, Walk Test, Recovery of Function, Genetic Therapy, and Medical and Health Sciences
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Spinal Muscular Atrophy is the most common genetic cause of infant death. Due to its severity, there is a need for methods for automated estimation of disease progression. In this paper we propose a Convolutional-Neural-Network (CNN)... more
Spinal Muscular Atrophy is the most common genetic cause of infant death. Due to its severity, there is a need for methods for automated estimation of disease progression. In this paper we propose a Convolutional-Neural-Network (CNN) model to estimate disease progression during infants’ natural behavior. With the proposed methodology, we were able to predict each child’s score on current behavior-based clinical exams with an average per-subject error of 6.96 out of 72 points (<10 % difference), using 30-second videos in leave-one-subject-out-cross-validation setting. When simple statistics were used over 30-second video-segments to estimate a score for longer videos, we obtained an average error of 5.95 (\(\sim \)8 % error rate). By showing promising results on a small dataset (N \(=\) 70, 2-minute samples, which were handled as 1487, 30-second video segments), our methodology demonstrates that it is possible to benefit from CNNs on small datasets by proper design and data handling choices.
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Managing the stresses of parenting a child with cerebral palsy (CP) can be challenging. This study sought to identify factors that are associated with higher levels of caregiver stress. A retrospective review of data from the Learn From... more
Managing the stresses of parenting a child with cerebral palsy (CP) can be challenging. This study sought to identify factors that are associated with higher levels of caregiver stress. A retrospective review of data from the Learn From Every Patient™ project conducted in an interdisciplinary CP clinic were used to compare caregiver responses on 2 subsets (financial and time/emotional) of the Assessment of Caregiver Experience in Neuromuscular Disorders (ACEND) and physical and medical characteristics of the child. The range of scores in both the financial and emotional subset was large. The presence of behavior problems, seizures, and severity of CP showed the strongest associations with emotional stress and accounted for 14% of the variance in scaled scores (r= 0.392, adj R2= 14.3, p&amp;amp;amp;amp;amp;amp;lt; 0.01). The child&amp;amp;amp;amp;amp;amp;#39;s age was not significantly related to parental stress. The most highly reported areas of stress were worry about the child&amp;amp;amp;amp;amp;amp;#39;s pain, and the financial impact of lost wages. Caregiver experience varied widely and is associated with a range of factors among families caring for a child with CP. Further research is needed to test whether interventions to minimize the areas of greatest stress could make a meaningful difference in family functioning.
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The ability to individualize recommendations or expectations of disease progression based on a patient's unique characteristics has merit for use in sporadic inclusion body myositis (sIBM). Fifty-five subjects with sIBM completed a... more
The ability to individualize recommendations or expectations of disease progression based on a patient's unique characteristics has merit for use in sporadic inclusion body myositis (sIBM). Fifty-five subjects with sIBM completed a battery of strength and functional outcomes at 2 study visits. These were used to develop mathematical models of disease progression in patients with sIBM for use in clinical and research settings. The six minute walk test (6MWT) distance declined an average of 27.5 meters (12%) per year. Significant factors that predict 6MWT were knee extension and plantarflexion strength and body weight, whereas the ability to stand from a chair was impacted by elbow extension strength. Stepping up on a curb was influenced by the patient's age at diagnosis and by knee extension. Statistical models to predict functional decline in sIBM were developed. Statistical models help explain the complex factors that influence decreased walking ability and other functional...
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Reference values for head shape do not currently exist making it difficult to consistently and accurately identify infants with deformational plagiocephaly (DP). To identify reference values for head shape for typically developing infants... more
Reference values for head shape do not currently exist making it difficult to consistently and accurately identify infants with deformational plagiocephaly (DP). To identify reference values for head shape for typically developing infants using a modified version of plagiocephalometry (PCM). Prospective cohort study METHODS: 134 typically developing infants four to nine months of age were recruited randomly from center-based daycares or via a convenience sample from in-home settings. A modified version of PCM was completed to assess head shape. The median values were as follows: ear difference (ED) =3 mm, anterior-sinistra minus anterior-dextra (ASAD) =3 mm, posterior-dextra minus posterior-sinistra (PDPS) =2 mm, oblique diameter difference (ODD) =3 mm, oblique diameter difference index (ODDI) =102.1%, and cranio proportional index (CPI) =84.8%. The 75(th) percentile (Q3) values were ED=6mm, ASAD=5 mm, PDPS=3 mm, ODD=5 mm, ODDI=103.5%, and CPI=88.3%. Values were not significantly di...
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To describe and report the effect of an 8-week individualized, progressive, treadmill training program on the ambulatory ability of a 4-year-old child with myelomeningocele without functional ambulation. Heart rate and speed on the... more
To describe and report the effect of an 8-week individualized, progressive, treadmill training program on the ambulatory ability of a 4-year-old child with myelomeningocele without functional ambulation. Heart rate and speed on the 2-minute walk test (2MWT) were used to individualize training. Ambulatory outcome measures taken at baseline, postintervention, and 6 weeks postintervention included the 2MWT and the Timed &amp;amp;amp;amp;amp;amp;quot;Up and Go&amp;amp;amp;amp;amp;amp;quot; (TUG). The Pediatric Evaluation of Disability Index was used to measure functional mobility. Improvements were noted in ambulatory ability after training (2MWT, 64.10%; TUG, 34.66%) and at the 6-week follow-up (2MWT, 58.97%; TUG, 34.24%). The Pediatric Evaluation of Disability Index showed no significant difference. Use of an individualized treadmill training program may have improved the ambulatory skills of a preschooler with myelomeningocele. More rigorous studies are needed to determine the effects of treadmill training with the spina bifida population and identify optimal training parameters.
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Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Subjects (n=91; 16.8 ± 4.5 years) were assessed at baseline and 6-month intervals for 2 years. We analyzed... more
Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Subjects (n=91; 16.8 ± 4.5 years) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤ 4 using repeated measures. Eight (12-33 years) died during the study. Sixty-six completed12-month follow-up; 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. FVC% predicted declined significantly only after 2 years. However, Brooke scale, Egen Klassifikation (EK) scale, elbow flexion and grip strength declined significantly over both 1 and 2 years. Brooke scale, EK scale, and elbow flexion and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affec...
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Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups resulting in a loss of independence and the need for assistive devices... more
Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups resulting in a loss of independence and the need for assistive devices and supportive care. To assess treatment benefit of new therapies, a patient-reported outcome measure of physical function was developed. The tool was rigorously developed in accordance with the U.S. Food and Drug Administration (FDA) Patient Report Outcomes (PRO) Guidance. A single-visit, observational study was conducted. Standard qualitative analytical methods were employed to analyze interview data and generate questionnaire items. Twenty concept elicitation and 19 cognitive debriefing interviews were conducted, and 6 expert physicians were consulted. The tool consists of 11 items scored on a 0-10 numerical rating scale. Subjects completed the questionnaire utilizing either paper or electronic administration. We have developed a PRO tool in alignment with FDA PRO Guidance for use in the functional assessment of treatment benefit in sIBM. This article is protected by copyright. All rights reserved.
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To assess self-reported physical functioning in patients with sporadic inclusion body myositis (sIBM), the sIBM Physical Functioning Assessment (sIFA) was developed. This research establishes the validity, reliability, and responsiveness... more
To assess self-reported physical functioning in patients with sporadic inclusion body myositis (sIBM), the sIBM Physical Functioning Assessment (sIFA) was developed. This research establishes the validity, reliability, and responsiveness of the sIFA in patients with sIBM. Data from 3 small, noninterventional, observational studies were analyzed. Several measures of physical function were included to assess validity. Reliability (Cronbach alpha, test-retest intraclass correlations), construct validity (correlations, analyses of variance), and responsiveness (effect size estimates) were evaluated. Cronbach alphas (range=0.86-0.91) and test-retest reliability (0.91) were highly satisfactory. Correlations with other measures provided evidence of convergent validity. sIBM patients able to walk without assistive devices scored significantly better on the sIFA (means=36.0-47.05) than those who require power mobility or wheelchairs (means=54.9-71.5), demonstrating the discriminating ability...
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Adolescent, Medicine, Humans, Child, Walking, and 12 moreMale, Alternative splicing, Duchenne Muscular Dystrophy, Clinical Sciences, Longitudinal Studies, Disease Progression, Alternative Splicing, Exercise Test, Neurosciences, Oligonucleotides, Mobility limitation, and Outcome assessment (Health care)
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DMD is a rare, degenerative, genetic disease that results in progressive muscle loss and premature death and affects 1:5000 male births. Clinically manifest pulmonary dysfunction often occurs when DMD patients become non-ambulant and is... more
DMD is a rare, degenerative, genetic disease that results in progressive muscle loss and premature death and affects 1:5000 male births. Clinically manifest pulmonary dysfunction often occurs when DMD patients become non-ambulant and is preceded by subclinical deterioration of pulmonary function tests (PFTs). Specifically, MIP and MEP % predicted deteriorate by approximately 4 % per year between the ages of 8–19. Eteplirsen is an investigational drug designed to enable functional dystrophin production in boys who are amenable to skipping exon 51. When dosed for up to 120 weeks, stabilization of 6 min walk distance was demonstrated in a Phase 2b study. 12 boys were randomized 1:1:1 to 30/50 mg/kg or placebo. Upon completion of a 24-week double-blind, placebo-controlled phase, all patients were enrolled in an open-label extension and the placebo-treated patients initiated eteplirsen treatment. FVC, FVC % predicted, MIP, MEP and MIP and MEP % predicted were assessed every 12 to 24 weeks. For all 12 patients changes in function at Week 120 were examined from Week 1, and from last assessment pre-eteplirsen administration (Week 1 for 8 patients and Week 24 for 4 patients). One-sample t-test was used for statistical analysis. Reported here are the results for all 12 patients, including two patients who became non ambulant by Week 24. Median age at Week 120 was 12 years. The 120 week data for 5 of the 6 PFT parameters were not statistically different from baseline, with the exception of a statistically significant increase in MEP. Furthermore, individual patient values for all 12 patients continue to be in the age-adjusted normal ranges indicative of continued normal pulmonary function without the need for any ventilation assistance or respiratory failure. A PFT update after 3 years of treatment (Week 144) will be provided. Eteplirsen dosed for up to 120 weeks demonstrated stability on PFT measures contrary to a steady decline expected in DMD patients of this age.
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The Americans with Disabilities Act of 1990 (ADA) is an important piece of civil rights legislation that can assist pediatric physical therapists in maximizing the opportunities afforded the children and adolescents they serve. The four... more
The Americans with Disabilities Act of 1990 (ADA) is an important piece of civil rights legislation that can assist pediatric physical therapists in maximizing the opportunities afforded the children and adolescents they serve. The four sections of the ADA with particular relevance to children and adolescents with disabilities are: employment, public and private accommodations, transportation, and telecommunications. An overview of the regulations governing each of these areas and examples relevant to children and adolescents will be given. An understanding of the provisions of the ADA will assist a therapist in designing a treatment plan that will help prepare children and adolescents with disabilities for their transition into leading productive, fulfilling lives as adults. This plan should include functionally based approaches to treatment, child and family education, and advocacy training. Knowledge of the provisions of the ADA and the implications for children with disabilities...
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The need for a valid and reliable measure of upper extremity (UE) function in Duchenne muscular dystrophy (DMD) is critical as current tools focus on distal ability, use an ordinal scale, and/or require significant training to use... more
The need for a valid and reliable measure of upper extremity (UE) function in Duchenne muscular dystrophy (DMD) is critical as current tools focus on distal ability, use an ordinal scale, and/or require significant training to use reliably. ACTIVE-seated was developed using the Microsoft Kinect platform. This camera system and gaming interface gathers UE positional data while providing the platform for video games to improve consistency of performance. Our purpose was to determine the validity and test–retest reliability of ACTIVE-seated as an UE tool in DMD. A sample of 40 subjects (mean age 13 ± 3) with DMD (Brooke level 1–5) and 15 controls were tested. Eleven subjects returned for next-day testing. ACTIVE-seated uses skeletal tracking to quantify the UE excursion in the X (left, right), Y (overhead), and Z (forward) planes. The functional reaching volume (FRV) of the area around the subject, encompassing both UE and trunk movement, was reported. The raw data were converted into a percent predicted based on the subject’s UE length to standardize comparisons between subjects and accommodate growth. One hundred percent of predicted would equal the accessible FRV of solely the UE without leaning by the trunk. The FRV of subjects with DMD ranged from 6% to 150% (mean: 69%) of predicted FRV, with controls all reaching well over 100% (mean: 147%). ACTIVE-seated discriminately ranked subjects with DMD by Brooke level and from controls (p
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Background/Objective DMD, a rare, degenerative, X-linked recessive genetic disease is mostly caused by frame-shift mutations in the dystrophin gene, preventing production of dystrophin protein. Eteplirsen, an investigational PMO designed... more
Background/Objective DMD, a rare, degenerative, X-linked recessive genetic disease is mostly caused by frame-shift mutations in the dystrophin gene, preventing production of dystrophin protein. Eteplirsen, an investigational PMO designed to enable production of internally truncated yet functional dystrophin in boys amenable to exon 51-skipping, was evaluated in previously conducted clinical studies (33, 28, and 201), and is currently being evaluated in ongoing clinical trials. Methods Twelve eligible boys aged 7–13 years were randomized 1:1:1 to eteplirsen 30/50 mg/kg/wk, or placebo for 24 weeks. All patients transitioned into an ongoing open-label extension trial at Week 25 with eteplirsen 30/50 mg/kg. Efficacy endpoints included 6MWT, PFT, and %-dystrophin positive fibers. Safety assessments included AE recording, ECG, ECHO, and safety laboratory testing. Results After 3.2 years of treatment, all patients previously evaluable on 6MWT (mITT; n=10) showed continued ambulation. A 65.4 meter treatment benefit (p≤0.017) on 6MWT through Week 168 was observed for patients in the eteplirsen treated cohort (n=6) compared with the placebo/delayed-treatment cohort (n=4). After declining on average 7.6 meters/month baseline through Week 36, the placebo/delayed-treatment cohort experienced a decline of 2.2 meters/month and the eteplirsen cohort a decline of 1.9 meters/month in the timeframes in which meaningful levels of dystrophin were likely produced (Week 36–168 for the Placebo/delayed-treatment cohort and Week 12–168 for the eteplirsen cohort). All 12 patients demonstrated PFT stability baseline through Week 168, including MIP (+11.1%, p=NS), MEP (+13.5%, p=NS), and MIP/MEP %-predicted (-2.4%/-6.3%, p=NS). No deaths, discontinuations, treatment-related SAEs, immune activation including infusion reactions, or clinically significant abnormal laboratory, ECG, or ECHO findings were reported. Conclusion The observed slowed decline in walking distance and sustained pulmonary function contrasts with rapid declines in ambulation and pulmonary capacity observed in DMD natural history of boys this age. Eteplirsen was well tolerated after 3.2 years of weekly infusions.
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We introduce a methodology and grammar for the description and development of therapeutic gestures using a natural user interface (NUI) device such as the Microsoft Kinect and provide some implementation guidelines for developing... more
We introduce a methodology and grammar for the description and development of therapeutic gestures using a natural user interface (NUI) device such as the Microsoft Kinect and provide some implementation guidelines for developing therapeutic games using these gestures. Gesture recognition is a widely studied area and the Kinect SDK provides skeletal tracking to assist in pose determination. However, most gesture recognition systems assume a rather pristine gesture. Noise in the system is filtered, but the underlying gesture is fairly robust. For Physical Therapy (PT) performed for individuals with neuromotor deficits, gestures need to allow for a wider spectrum of possible motion paths. For instance, following a stroke that results in hemiparesis, individuals often display patterns of motor compensation, thus altering the trajectories of many motor movements. This has required a rethinking of how gestures are defined and the development of a more analytical and simplistic formulatio...
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Current trials in Duchenne muscular dystrophy (DMD) have focused on the distance traveled using the 6-min walk test (6MWT). As a primary efficacy outcome for clinical trials it can be quantified over a continuous scale. Limitations... more
Current trials in Duchenne muscular dystrophy (DMD) have focused on the distance traveled using the 6-min walk test (6MWT). As a primary efficacy outcome for clinical trials it can be quantified over a continuous scale. Limitations include a self-selected walking speed irrespective of encouragement and weakness predisposing to falls. We predicted that a monetary incentive of $50 could improve same-day 6MWT performance. Nine DMD boys (mean age 7.7) were instructed to walk quickly, not run. Encouragement was permitted throughout the test. Subjects were then stratified into an above or below 350 m group and randomly assigned to either the incentivized (IN) or non-incentivized (non-IN) group. A second 6MWT was completed after a rest period during which the following instructions were provided. The IN was told “If you walk faster on this repeat 6MWT you will receive $50. Remember to walk quickly and safely.” The non-IN were told “You are repeating the 6MWT and remember to walk quickly and safely.” The same evaluator conducted both tests, was blinded to group, and provided the same encouragement during each 6MWT. Current data is available in 9 boys, however data on a cohort of 36 will be presented. The boys in the IN (n = 5, mean age = 7.7) walked an average of 44 m (11%, max: 105 m) farther on the second 6MWT. The non-IN (n = 4, mean age = 7.6) walked an average of 5.1 m (2%, max: 16 m) farther. The increase in distance in IN was most distinct in the above 350 m (75 m or 17%) compared to the below 350 m (24 m or 7%) group. Our pilot data suggest that a financial incentive results in a measurable increase in within-day 6MWT distance in DMD. This variance is more pronounced in the above 350 m group which is the typical target for clinical trials. This data suggests that external motivators have a significant impact on the 6MWT and should be considered in the design and interpretation of clinical trials.
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Children with cerebral palsy frequently receive therapeutic intervention to remediate standing balance deficits. Evaluation of the impairments associated with poor balance could facilitate more effective treatment programs. This study... more
Children with cerebral palsy frequently receive therapeutic intervention to remediate standing balance deficits. Evaluation of the impairments associated with poor balance could facilitate more effective treatment programs. This study evaluated the relationship between lower extremity force production, range of motion and standing balance in thirty-five children between the ages of 6 and 14 years of age with spastic cerebral palsy. Standing balance was evaluated using the Pediatric Clinical Test of Sensory Interaction (P-CTSIB). Hand-held dynamometry was used to assess force production and goniometry was used to assess range of motion. The results indicated that force production and range of motion are highly related to standing balance. Blocked, hierarchical multiple regression analysis revealed that force production explained 41% of the variance in P-CTSIB scores in this sample, while range of motion explained an additional 13%. Therefore, the total variance explained by these variables was 54%. Results of this study suggest that impairment level testing may allow the development of more effective individualized intervention programs to remediate balance deficits. Clinical suggestions are provided.
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Cerebral Palsy, Adolescent, Physical Therapy, Humans, Child, and 15 moreIntervention, Female, Male, Muscles, Balance, Motion, Clinical Sciences, Physical Fitness, Multiple regression analysis, Muscle Force, Postural Balance, Lower Extremity, Child preschool, Muscular strength, and Paediatrics and reproductive medicine
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ABSTRACT
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To describe and report the effect of an 8-week individualized, progressive, treadmill training program on the ambulatory ability of a 4-year-old child with myelomeningocele without functional ambulation. Heart rate and speed on the... more
To describe and report the effect of an 8-week individualized, progressive, treadmill training program on the ambulatory ability of a 4-year-old child with myelomeningocele without functional ambulation. Heart rate and speed on the 2-minute walk test (2MWT) were used to individualize training. Ambulatory outcome measures taken at baseline, postintervention, and 6 weeks postintervention included the 2MWT and the Timed &amp;amp;amp;amp;amp;amp;quot;Up and Go&amp;amp;amp;amp;amp;amp;quot; (TUG). The Pediatric Evaluation of Disability Index was used to measure functional mobility. Improvements were noted in ambulatory ability after training (2MWT, 64.10%; TUG, 34.66%) and at the 6-week follow-up (2MWT, 58.97%; TUG, 34.24%). The Pediatric Evaluation of Disability Index showed no significant difference. Use of an individualized treadmill training program may have improved the ambulatory skills of a preschooler with myelomeningocele. More rigorous studies are needed to determine the effects of treadmill training with the spina bifida population and identify optimal training parameters.
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To present an algorithm with accompanying treatment parameters for the management of congenital muscular torticollis (CMT) based on the best available literature. A systematic search of PubMed, MEDLINE, CINHAL, and Cochrane databases was... more
To present an algorithm with accompanying treatment parameters for the management of congenital muscular torticollis (CMT) based on the best available literature. A systematic search of PubMed, MEDLINE, CINHAL, and Cochrane databases was conducted to identify evidence to guide the conservative management of CMT. An evidence-based algorithm was created based on three prognostic factors that influence treatment duration and outcome, including a sternocleidomastoid fibrotic mass, passive range of motion rotation deficit, and age at initiation of treatment. Preliminary treatment parameter recommendations for clinic and home programming accompany the algorithm. Use of the proposed evidence-based algorithm with accompanying preliminary treatment parameter recommendations may improve consistency of care and outcomes for infants with CMT. While a higher level of evidence supports the three prognostic factors utilized in the algorithm, research gaps continue to exist with regards to treatment parameters.
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Clinical trials are being conducted for DMD using timed walking as a primary outcome measure due to the ease of administration, ability to quantify distance, and established validity. Unfortunately, participation in clinical trials is not... more
Clinical trials are being conducted for DMD using timed walking as a primary outcome measure due to the ease of administration, ability to quantify distance, and established validity. Unfortunately, participation in clinical trials is not equally accessible to all individuals due to the lack of a reliable, valid and sensitive upper extremity measure for the non-ambulatory population. It is essential that this gap in knowledge be filled, as upper body movement is required for many activities of daily living impacting quality of life across the lifespan and abilities in individuals with DMD. The purpose of this project was to develop a reliable and engaging upper extremity measure to quantify workspace volume, reaching velocity, and rate of arm fatigue using the Kinect system to capture data during a video virtual reality game across the spectrum of abilities. The Microsoft Kinect Windows 7 used for this project is a controller-free gaming device that projects an infrared reference ma...
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Evaluate the utility of timed walking tests in DMD. Many different timed walking tests have been shown to be accurate, reproducible, and simple to administer (10 m walk (10 mw), 2 min (2MWT), and 6 min (6MWT)). Recently the 6MWT has... more
Evaluate the utility of timed walking tests in DMD. Many different timed walking tests have been shown to be accurate, reproducible, and simple to administer (10 m walk (10 mw), 2 min (2MWT), and 6 min (6MWT)). Recently the 6MWT has emerged as the gold standard for use in clinical trials. Thirty five subjects with DMD were evaluated 95 times. Tests include walking distance; isometric quadriceps strength testing and the North Star Ambulatory Assessment (NSAA). Mixed model analysis revealed that both the 2MWT and 6MWT are highly significant correlations to the NSAA (p < 0.0001) and quadriceps strength (p = 0.05). Boys walked a consistent speed throughout the entire test. Velocity during each minute of a single session ranged from 0.97 to 1.06 m/s, showing a high degree of correlation at each timed interval (r = 0.725 − 0.921; p < 0.001). Wilcoxon signed rank tests on walking test group data revealed no significant difference between distances on day 1 and day 2 for either the 2M...
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Introduction: The correlation of strength with performance on functional outcomes has not been evaluated in Becker muscular dystrophy (BMD), yet the determination of proper outcome measures is critical to the success of upcoming trials... more
Introduction: The correlation of strength with performance on functional outcomes has not been evaluated in Becker muscular dystrophy (BMD), yet the determination of proper outcome measures is critical to the success of upcoming trials designed to improve strength. Methods: Lower extremity strength and performance on functional outcome measures were tested in 25 ambulatory subjects with BMD. Results: All subjects demonstrated marked knee extensor and flexor muscle weakness. Knee extensor strength was correlated with performance on the Berg Balance Scale and stair climbing. Knee flexor strength was highly correlated with performance on all functional outcomes, including timed walking distances. Conclusions: This profile differs from that previously reported in other neuromuscular diseases and demonstrates the importance of designing outcome measures for clinical trials in muscle disorders with considerations for the disease process, the mode, and the target of intervention. The findi...
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ABSTRACT The evidence for Constraint-Induced Movement Therapy (CIMT) effectiveness for infants and toddlers with unilateral cerebral palsy is minimal. We performed a pilot study of CIMT using one-month usual care, one-month intervention,... more
ABSTRACT The evidence for Constraint-Induced Movement Therapy (CIMT) effectiveness for infants and toddlers with unilateral cerebral palsy is minimal. We performed a pilot study of CIMT using one-month usual care, one-month intervention, and one-month maintenance (return to usual care) phases on five infants (7- to 18-month old). For the CIMT phase, the infants received 2 hr of occupational therapy and 1 hr of parent-implemented home program for five days/week. The infants were casted for the first 23 days, and bimanual therapy was provided for the last three days. Fine motor skills for the more affected arm and gross motor skills improved significantly during the CIMT; these gains were maintained at one-month follow-up. Individual infant data show mixed effects. This pilot study provides initial evidence that CIMT is feasible for infants with unilateral cerebral palsy, and presents preliminary data for CIMT on fine and gross motor performance.
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Sporadic inclusion body myositis causes progressive functional loss due to declining muscle strength. Although the underlying cause is unknown, clinical trials are underway to improve strength and function. Selection of appropriate... more
Sporadic inclusion body myositis causes progressive functional loss due to declining muscle strength. Although the underlying cause is unknown, clinical trials are underway to improve strength and function. Selection of appropriate outcome measures is critical for the success of these trials. The 6-min walk test has been the de facto standard for assessing function in neuromuscular disease; however, the optimal walking test has not been determined in this disease. In this study, 67 individuals with sporadic inclusion body myositis completed a battery of quantitative strength and functional tests including timed walking tests, patient-reported outcomes, and other tasks. The 2-min and 6-min walk tests are highly correlated to each other (r=0.97, p<0.001) and to all lower extremity strength, patient-reported, and functional measures in this population. All subjects completed the 2-min walk test, but 7% of subjects were unable to walk the full 6-min of the 6-min walk test due to fati...