Rasch analysis of the 32-item motor function measure in ambulant patients with Duchenne muscular dystrophy.

OBJECTIVE: This study analyzed the 32-item Motor Function Measure in a cohort of ambulatory patients with Duchenne muscular dystrophy using Rasch measurement methods. DESIGN: This is a psychometric study. SETTING: Rehabilitation centre of a large public children's hospital in Shenzhen, China. PARTICIPANTS: Data from 176 genetically confirmed ambulant patients with Duchenne muscular dystrophy (mean age 7.3 years, SD 2.3 years, range 3.1-13.1 years) were analyzed. RESULTS: Rasch analyses supported the Motor Function Measure domain D1 as a reliable (person reliability = 0.88, person separation index = 2.71) and valid (acceptable targeting, little misfit, minimal category disordering) measure in ambulant patients with Duchenne muscular dystrophy. Remodelling the domain D1 by collapsing item 25 from 4 to 3 response categories addressed the problematic disordered thresholds, resulting in a rebuilt domain D1 with enhanced measurement properties. However, findings for domains D2 and D3 did not fulfil most Rasch model expectations. There were disordered thresholds for most items in domains D2 and D3, with low reliability coefficients, item mistargeting and misfit, and large ceiling effects. CONCLUSION: Rasch analyses confirmed that the Motor Function Measure domain D1 was reliable and valid and provided a unidimensional measure for motor function in ambulant Duchenne muscular dystrophy patients. Accuracy of measurement had been enhanced through remodelling, and a rebuilt domain D1 with category collapsing for item 25 was proposed. The analysis revealed multiple limitations of the domains D2 and D3 that certain essential psychometrics were poorly met and, therefore, should be used with caution in this patient group.

Impact of the COVID-19 pandemic on neuromuscular rehabilitation setting. Part 2: patients and families' views on the received health care during the pandemic.

This study explored views of users with muscular dystrophies and their caregivers on staff-user relationships and the treatments provided by a Rehabilitation Centre during the pandemic. Patients and relatives were asked to anonymously complete an open-ended questionnaire exploring their views on these aspects. Fifty-four patients and 40 caregivers gave their informed consent and participated in the survey. Fifty-three patients were adults, 28% suffering from Duchenne/Becker muscular dystrophy. Patients reported 269 comments on health care services provided during the pandemic, 132 (49%) concerning positive aspects and 137 (51%) negative aspects. The prompt restart of the rehabilitation therapies and the staff closeness over the pandemic were the practical aspects most frequently appreciated (46.9%), while closer family contacts and the perception of being able to rely on the Centre's constant support were the most cited psychological aspects (53.1%). Architectural barriers, difficulties in accessing public health services, economic difficulties, and lack of support from welfare and other agencies were the practical critical points most frequently reported (89%). In addition, social isolation, and loneliness due to fear of contagion were the most negative psychological aspects (10.1%). As regard the caregivers' views, participants reported 151 comments. Of these, 86 (56.9%) were positive and 65 (43.1%) were negative. Among the positive aspects, the psychological ones - such as closer family contacts, not feeling abandoned and counting on the constant Centre's professional support prevailed (53.5%). As for the negative aspects, most caregivers (92.6%) believe that the pandemic exacerbated their financial and bureaucratic difficulties, particularly in poorer families.

Necesidad de rehabilitación física en un paciente con distrofia muscular progresiva de Duchenne / Necessity of physical rehabilitation in a patient with Duchenne progressive muscular dystrophy

Se presenta el caso clínico de un adolescente de 18 años de edad, atendido en el Policlínico Docente Armando García Aspurú de Santiago de Cuba por presentar antecedentes de retardo en el desarrollo psicomotor, marcha anadeante, caídas frecuentes, signo de Gowers positivo, fuerza muscular proximal disminuida en miembros superiores e inferiores, pseudohipertrofia de los gemelos, atrofia de cuádriceps pectoral y escapular. Los signos y síntomas clínicos permitieron diagnosticar una distrofia muscular de Duchenne. Como no pudo realizarse el tratamiento rehabilitador necesario, se produjo un deterioro músculo - esquelético progresivo y severo del paciente.

The case report of an 18 years adolescent is presented. He was assisted at Armando García Aspurú Teaching Polyclinic in Santiago de Cuba due to a history of psychomotor development retardation, wandering march, frequent falls, positive Gowers sign, diminished proximal muscular force in upper and lower limbs, twins pseudohypertrophy, atrophy of pectoralis and scapular quadriceps. The clinical signs and symptoms allowed to diagnose a Duchenne muscular dystrophy. As the necessary rehabilitative treatment could not be carried out, a progressive and severe musculoskeletal deterioration of the patient took place.

Feasibility and effectiveness of a novel dynamic arm support in persons with spinal muscular atrophy and duchenne muscular dystrophy.

BACKGROUND: Neuromuscular disorders (NMD) commonly affect the upper extremity. Due to muscle weakness, performance of daily activities becomes increasingly difficult, which leads to reduced independence and quality of life. In order to support the performance of upper extremity tasks, dynamic arm supports may be used. The Yumen Arm is a novel dynamic arm support specially developed for people with NMD. The aim of this study is to evaluate the feasibility and effectiveness of the Yumen Arm in persons with Duchenne Muscular Dystrophy (DMD) and persons with Spinal Muscular Atrophy (SMA). METHODS: Three persons with DMD and three persons with SMA participated in this study. All participants conducted a set of measures with and without the Yumen Arm. Outcome measures were: active range of motion of the arm and trunk (i.e. Reachable Workspace, Functional Workspace, and trunk movement), fatigue (OMNI-RPE), Performance of Upper Limb (PUL) scale and some additional activities of daily living. User experiences were collected using a questionnaire. RESULTS: The Yumen Arm could be used by all participants. Results showed a median increase in active range of motion (4% relative surface area), and a median increase of function ability (> 11% PUL score) when using the Yumen Arm. In addition, three out of four (data from 2 participants was missing) participants indicated that activity performance was less fatiguing when using the Yumen Arm. Four out of five (data from 1 participant was missing) participants indicated that they would like to use the Yumen Arm in their daily lives. CONCLUSION: This study is one of the first studies describing a range of objective measures to examine the effectiveness of a dynamic arm support. Based on these measurements we can conclude that the Yumen Arm effectively improves arm function in NMD patients, however the effectiveness varies a lot between individual subjects. We provided detailed recommendations for the improvement of the Yumen Arm, and possible also for the development of other dynamic arm supports. This study showed a lot of variability between individual subjects, which emphasizes the importance of tuning dynamic arm supports based on individual user characteristics, such as scoliosis, functional capacity and muscle strength.

Safety, feasibility, and efficacy of strengthening exercise in Duchenne muscular dystrophy.

BACKGROUND: This two-part study explored the safety, feasibility, and efficacy of a mild-moderate resistance isometric leg exercise program in ambulatory boys with Duchenne muscular dystrophy (DMD). METHODS: First, we used a dose escalation paradigm with varying intensity and frequency of leg isometric exercise to determine the dose response and safety in 10 boys. Second, we examined safety and feasibility of a 12-wk in-home, remotely supervised, mild-moderate intensity strengthening program in eight boys. Safety measures included T2 MRI, creatine kinase levels, and pain. Peak strength and function (time to ascend/descend four stairs) were also measured. RESULTS: Dose-escalation revealed no signs of muscle damage. Seven of the eight boys completed the 12-wk in-home program with a compliance of 84.9%, no signs of muscle damage, and improvements in strength (knee extensors P < .01; knee flexors P < .05) and function (descending steps P < .05). CONCLUSIONS: An in-home, mild-moderate intensity leg exercise program is safe with potential to positively impact both strength and function in ambulatory boys with DMD.

Kinesiology Taping in Duchenne Muscular Dystrophy: Acute Effects on Performance, Gait Characteristics, and Balance.

AIM: This study was aimed to investigate the acute effects of kinesiology taping (KT) on physical performance, gait characteristics, and balance in early-stage Duchenne Muscular Dystrophy (DMD). METHOD: Forty-five children at early functional level of DMD were included. 6-minute walk test (6MWT), and timed performance tests were performed; gait characteristics, and balance were assessed before and one hour after taping. KT was applied to bilateral quadriceps and tibialis anterior muscles. The comparison of assessments was performed by using Wilcoxon Signed Ranks test. RESULTS: Significant increase in the distance of 6MWT, decrease in the duration of descending 4 steps, and 10 m walk timed performance tests, improvements in all of the gait characteristics, and balance were determined after taping (p < .05). CONCLUSIONS: KT has positive acute effects on performance and gait of children with DMD at early functional level which encourages therapists to use KT as a complementary approach in rehabilitation programs.

Rehabilitación respiratoria para pacientes con distrofia muscular de Duchenne en etapas de pérdida de la marcha / Respiratory rehabilitation for patients with Duchenne muscular dystrophy in stages of loss of gait

Duchenne muscular dystrophy (DMD) is one of the most common neuromuscular diseases. Its evolution with well-defined stages related to motor and functional alterations, allows easily establishing relationships with respiratory function through a simple laboratory assessment including vital capacity (VC) measurements as well as peak cough flows. Without any treatment with respiratory rehabilitation, the main cause of morbidity and mortality is ventilatory failure, secondary to respiratory pump muscles weakness and inefficient cough. The VC plateau is reached during the non-ambulatory stages, generally after 13 years old. Respiratory rehabilitation protocols, including air stacking techniques, manual and mechanical assisted coughing and non-invasive ventilatory support, can effectively addressed the VC decline as well as the decrease in peak cough flows, despite advancing to stages with practically non-existent lung capacity. Non-invasive ventilatory support may be applied after 19 years old, initially at night and then extending it during the day. In this way, survival is prolonged, with good quality of life, avoiding ventilatory failure, endotracheal intubation and tracheostomy. This article proposes staggered interventions for respiratory rehabilitation based on the functional stages expected in the patient with DMD who has lost ambulation.

La distrofia muscular de Duchenne (DMD) es una de las enfermedades neuromusculares más frecuentes. Su curso evolutivo con etapas de declinación en la funcionalidad motora bien definidas, permite fácilmente establecer relaciones con la función respiratoria a través de un laboratorio de evaluación sencilla, básicamente de la capacidad vital (CV) y la capacidad tusígena. Sin intervenciones en rehabilitación respiratoria, la principal causa de morbimortalidad es la insuficiencia ventilatoria secundaria a debilidad de músculos de la bomba respiratoria e ineficiencia de la tos. En las etapas no ambulantes, se alcanza la meseta de la CV, generalmente después de los 13 años, su declinación junto con la disminución de la capacidad tusígena puede ser enfrentada efectivamente con la utilización de protocolos de rehabilitación respiratoria. Estos deben considerar la restitución de la CV con técnicas de insuflación activa o apilamiento de aire, tos asistida manual y mecánica, más soporte ventilatorio no invasivo, inicialmente nocturno después de los 19 años y luego diurno, pese a avanzar a etapas con capacidad pulmonar prácticamente inexistente. De esta manera, se prolonga la sobrevida, con buena calidad de vida, evitando el fallo ventilatorio, eventos de intubación endotraqueal y traqueostomía. Este artículo, hace propuestas escalonadas de intervención en rehabilitación respiratoria basadas en las etapas funcionales esperables en el paciente con DMD que ha perdido la capacidad de marcha.

Evaluation of an exercise-enabling control interface for powered wheelchair users: a feasibility study with Duchenne muscular dystrophy.

BACKGROUND: Powered wheelchairs are an essential technology to support mobility, yet their use is associated with a high level of sedentarism that can have negative health effects for their users. People with Duchenne muscular dystrophy (DMD) start using a powered wheelchair in their early teens due to the loss of strength in their legs and arms. There is evidence that low-intensity exercise can help preserve the functional abilities of people with DMD, but options for exercise when sitting in a powered wheelchair are limited. METHODS: In this paper, we present the design and the feasibility study of a new version of the MOVit device that allows powered-wheelchair users to exercise while driving the chair. Instead of using a joystick to drive the wheelchair, users move their arms through a cyclical motion using two powered, mobile arm supports that provide controller inputs to the chair. The feasibility study was carried out with a group of five individuals with DMD and five unimpaired individuals. Participants performed a series of driving tasks in a wheelchair simulator and on a real driving course with a standard joystick and with the MOVit 2.0 device. RESULTS: We found that driving speed and accuracy were significantly lowered for both groups when driving with MOVit compared to the joystick, but the decreases were small (speed was 0.26 m/s less and maximum path error was 0.1 m greater). Driving with MOVit produced a significant increase in heart rate (7.5 bpm) compared to the joystick condition. Individuals with DMD reported a high level of satisfaction with their performance and comfort in using MOVit. CONCLUSIONS: These results show for the first time that individuals with DMD can easily transition to driving a powered wheelchair using cyclical arm motions, achieving a reasonable driving performance with a short period of training. Driving in this way elicits cardiopulmonary exercise at an intensity found previously to produce health-related benefits in DMD.

Transition from Childhood to Adulthood in Patients with Duchenne Muscular Dystrophy.

Recently, progress has been observed in the knowledge about Duchenne Muscular Dystrophy (DMD), which is a severe and commonly diagnosed genetic myopathy in childhood, historically resulting in early death. Currently, there are a lot of methods available to improve the clinical course of DMD and extend patients' life expectancy to more than 30 years of age. The key issue for DMD patients is the period between 16-18 years of age, which is described as a transition from pediatric- to adult-oriented healthcare. Adolescents and adults with DMD have highly complex healthcare needs associated with long-term steroid usage, orthopedic, ventilation, cardiac, and gastrointestinal problems. The current paper provides a comprehensive overview of special healthcare needs related to the transfer of a patient with DMD from child-oriented to adult-oriented care. Additionally, the need to organize effective care for adults with DMD is presented.

Does Whole-Body Vibration Treatment Make Children's Bones Stronger?

PURPOSE OF REVIEW: To summarize the last 10 years of literature regarding the effects of whole-body vibration (WBV) on bone in children, and if WBV results in increased bone acquisition. RECENT FINDINGS: WBV intervention appears to be a safe intervention with beneficial effects on bone mass in some diseases and syndromes, but there is still low evidence for WBV in clinical practice. The positive effects on muscle strength, balance, and walking speed are more conclusive. One of the takeaways of this review is that well-trained individuals may not further improve bone mass with WBV; thus, interventions are more beneficial in pediatric individuals with Down syndrome or severe motor disabilities with low bone mass and reduced activity levels. WBV appears to be a safe non-pharmacological anabolic approach to increase bone mass in some pediatric populations; however, longer (> 6 months) and larger prospective studies are needed to elucidate the efficacy of WBV on bone health in young individuals.

Facilitators and Barriers to Wearing Hand Orthoses by Adults with Duchenne Muscular Dystrophy: A Mixed Methods Study Design.

BACKGROUND: To retard shortening of finger flexors in patients with Duchenne muscular dystrophy (DMD), hand orthoses are prescribed. However, many patients do not wear the orthoses regularly. To optimize orthotic interventions, we need insight into the factors influencing compliance. OBJECTIVE: To evaluate the compliance regarding hand orthoses in an adult DMD population and to explore experiences and perceptions of DMD patients wearing orthoses, and of their caregivers. METHODS: Mixed methods observational study, combining quantitative and qualitative data from medical charts combined with qualitative semi-structured interviews using a constant comparative method and a short validated questionnaire (D-QUEST). RESULTS: 65 medical charts were analyzed. 48 patients were assessed as needing hand orthoses, of whom 37.5 % were compliant. Qualitative data analyses revealed (1) motivation: preservation of hand function; (2) barriers: discomfort and impediments; (3) facilitators: good fit and personalized wearing schedule; (4) fitting process: satisfactory, but patients do not readily seek help when barriers appear. CONCLUSIONS: Patients are motivated to wear hand orthoses, but often discontinue use because of orthosis-and disease-specific barriers. The identification of these barriers leads to practical and feasible recommendations concerning the orthoses and the fitting process, such as less rigid material, preservation of some function while wearing the orthoses, and fixed evaluation points. The findings were confirmed by the D-QUEST.

Rehabilitation Following Fracture in Dystrophinopathy, A Case Series.

BACKGROUND: Boys with dystrophinopathies (DMD) are at increased risk of low bone mineral density and fracture. Femoral fracture is the most common extremity fracture and is accompanied by significant risk of functional loss. Care considerations for DMD have stressed that aggressive management may be needed to maintain ambulation and that surgical fixation allows early mobilization. OBJECTIVES: Describe 5 cases of femoral fracture in ambulatory boys with DMD and the course of care undertaken to optimize function. PATIENTS: Five boys with DMD median age 15y (12-16) who were independently ambulatory. Median 10m walk speed prior to their first fracture was 8 sec (7-17.37) and 4 of 5 were less than the 9 seconds predictive of 2 year ambulation retention. Three of the cases had a single incident causing fracture; the remaining cases had 2 and 3 incidents respectively representing a total of 8 fractures 6 of which were surgically stabilized. RESULTS: Following the first fracture, all 5 subjects regained some form of ambulation. Three patients regained independent ambulation and 2 with hand held support or contact guard. Two subjects went on to have additional falls with associated fracture. No patient regained the ability to rise from the floor and only one of the 5 regained the ability to climb steps and all demonstrated a decline in walking speed. CONCLUSION: Prompt orthopedic intervention, early mobility, and intensive rehabilitation even in the end stage ambulatory patient, were factors in helping preserve function in these patients with dystrophinopathies.

Evaluation of admittance control as an alternative to passive arm supports to increase upper extremity function for individuals with Duchenne muscular dystrophy.

The degree of upper extremity active range of motion provided by an admittance control robot compared with a commercially available passive arm support for individuals with DMD who have limited arm function was investigated in this study. The reachable workspace evaluation was used to assess active range of motion provided by both devices. A visual analog scale was also used to secure participant-reported outcome measures. The admittance control robot significantly increased reachable surface area scores compared with the passive arm support for the dominant arm (Wilcoxon T = 5, P = .022, r2 = 0.263) and for the nondominant arm (paired-samples t test, t(9) = 4.66, P = .001, r2 = 0.71). The admittance control robot also significantly decreased participant-reported exertion compared with the passive arm support. Results of this study substantiated the benefits of admittance control for individuals with DMD compared with a commercially available passive arm support.

Fisioterapia aquática em indivíduos com distrofia muscular: uma revisão sistemática do tipo escopo / Fisioterapia acuática en individuos con distrofia muscular: una revisión sistemática de alcance / Aquatic physical therapy in individuals with muscular dystrophy: systematic scoping review

RESUMO O objetivo deste estudo foi mapear o uso da fisioterapia aquática em indivíduos com distrofias musculares, de forma a caracterizar as intervenções no meio aquático e identificar componentes mensurados (variáveis estudadas e instrumentos utilizados nos estudos). A revisão sistemática do tipo de escopo incluiu estudos experimentais, descritivos e observacionais (em inglês, português e espanhol). As buscas foram realizadas nas plataformas Medline (PubMed), CINAHL, Embase, PEDro, Lilacs, ERIC, Scopus, Web of Science e Google Scholar. Os dados extraídos foram alocados em três categorias: (1) caracterização dos registros, (2) informações referentes a fisioterapia aquática e (3) componentes mensurados. Foram encontrados 556 registros e, destes, selecionados 20. As amostras dos estudos selecionados incluíram, na maioria, indivíduos com distrofia muscular de Duchenne, com idade entre 5 e 22 anos, que fizeram fisioterapia aquática com duração média de 45 minutos uma ou duas vezes por semana, por 21 semanas. Essas características corroboram estudos feitos em diferentes populações. A maioria dos estudos investigou alterações pulmonares e controle postural/desempenho funcional, poucos avaliaram os efeitos no sistema cardíaco. Recomenda-se usar a Egen Klassifikation, a North Star Ambulatory Assessment e fazer o teste de caminhada de seis minutos.

RESUMEN El presente estudio tuvo el objetivo de mapear la práctica de fisioterapia acuática por individuos con distrofias musculares, para caracterizar las intervenciones en el medio acuático e identificar los componentes medidos (variables estudiadas e instrumentos utilizados en los estudios). La revisión sistemática de alcance incluyó estudios experimentales, descriptivos y observacionales (en inglés, portugués y español). Se llevaron a cabo las búsquedas en Medline (PubMed), CINAHL, Embase, PEDro, Lilacs, ERIC, Scopus, Web of Science y Google Scholar. Los datos obtenidos se asignaron en tres categorías: (1) caracterización de registros; (2) informaciones sobre fisioterapia acuática; y (3) componentes medidos. Se encontraron 556 registros, de los cuales se seleccionaron 20. Las muestras de los estudios seleccionados incluyeron mayoritariamente a individuos con distrofia muscular de Duchenne, con edades entre 5 y 22 años, y que se habían sometido a sesiones de fisioterapia acuática con un promedio de duración de 45 minutos, una o dos veces por semana, durante 21 semanas. Estas características confirman estudios realizados con diferentes poblaciones. La mayoría de los estudios han investigado las alteraciones pulmonares y el control postural/rendimiento funcional, pero pocos han evaluado los efectos sobre el sistema cardíaco. Se recomienda emplear la Egen Klassifikation, la North Star Ambulatory Assessment y aplicar la prueba de caminata de seis minutos.

ABSTRACT The aim of this study is to map the use of aquatic physical therapy in individuals with muscular dystrophy, to characterize aquatic physical therapy intervention and identify measured components (variables and measurement instruments used) by the studies. A systematic scoping review included experimental, descriptive and observational studies (in English, Portuguese and Spanish languages). The searches were carried out on MEDLINE (PubMed), CINAHL, Embase, PEDro, Lilacs, ERIC, Scopus, Web of Science, Google Scholar. The extracted data were characterized into three categories: (1) characterization of the records, (2) information referring to aquatic physical therapy, and (3) measured components. There were 556 studies records and 20 records were selected. The studies samples included mostly individuals with Duchenne muscular dystrophy, aged between 5 and 22 years old. Aquatic physical therapy sessions lasted about 45 minutes, and one or two sessions per week were carried out for 21 weeks. That corroborates studies conducted in different populations. Most of the studies investigated pulmonary system and postural control/ functional ability, and a few studies evaluated cardiac system. Egen Klassifikation and North Star Ambulatory Assessment are recommended, and also to perform 6-minute walk test.

Ankle bracing practices in ambulatory, corticosteroid-naive boys with Duchenne muscular dystrophy.

INTRODUCTION: Loss of ambulation in Duchenne muscular dystrophy presages scoliosis, respiratory failure, and death. Strategies to maintain ankle range of motion are employed, but little evidence exists to support these approaches and limited information is available concerning current practice. METHODS: In this study we assessed baseline bracing data from 187 boys participating in a multicenter, international clinical trial. RESULTS: Ankle-foot orthoses (AFOs) were recommended for 54% of the boys, with nighttime static AFOs and nighttime dynamic AFOs utilized in 94% and 6% of these boys, respectively. Daytime static AFOs were recommended for 3 boys. Compliance with bracing recommendations was 54% for nighttime static braces and 67% for nighttime dynamic braces. DISCUSSION: The basis for the variation in recommended AFO use is unknown and requires further study. Long-term follow-up of boys may permit assessment of the effects of AFO use.

A Case Study With Symbihand: An sEMG-Controlled Electrohydraulic Hand Orthosis for Individuals With Duchenne Muscular Dystrophy.

With recent improvements in healthcare, individuals with Duchenne muscular dystrophy (DMD) have prolonged life expectancy, and it is therefore vital to preserve their independence. Hand function plays a central role in maintaining independence in daily living. This requires sufficient grip force and the ability to modulate it with no substantially added effort. Individuals with DMD have low residual grip force and its modulation is challenging and fatiguing. To assist their hand function, we developed a novel dynamic hand orthosis called SymbiHand, where the user's hand motor intention is decoded by means of surface electromyography, enabling the control of an electrohydraulic pump for actuation. Mechanical work is transported using hydraulic transmission and flexible structures to redirect interaction forces, enhancing comfort by minimizing shear forces. This paper outlines SymbiHand's design and control, and a case study with an individual with DMD. Results show that SymbiHand increased the participant's maximum grasping force from 2.4 to 8 N. During a grasping force-tracking task, muscular activation was decreased by more than 40% without compromising task performance. These results suggest that SymbiHand has the potential to decrease muscular activation and increase grasping force for individuals with DMD, adding to the hand a total mass of no more than 241 g. Changes in mass distributions and an active thumb support are necessary for improved usability, in addition to larger-scale studies for generalizing its assistive potential.

[Study of medical management for patients with Duchenne muscular dystrophy in Japan: what was changed by a practical guideline].

In order to clarify the changes in the medical management of patients with Duchenne muscular dystrophy after publication of a practical guideline, questionnaire surveys targeting Japanese certified neurologists and child neurologists before and four years after publication of the guideline were conducted. The practical guideline was evaluated by the majority of specialists as their preferred information source. After the guideline, a more aggressive trend was seen in steroid treatment, rehabilitation, regular assessment of spinal deformity, etc., and significant differences were observed in respiratory care and nutritional management. Although there were concerns that it would be hard to produce useful guidelines due to the lack of high-level evidence for a rare disease, guidelines including expert recommendations have proved to be an important tool for disseminating best practicable care. We are planning to revise the guideline based on the results of the analysis.

The Effect of Inspiratory Muscle Training on Duchenne Muscular Dystrophy: A Meta-analysis.

PURPOSE: This study reports the respiratory muscle training effect on strength and endurance in individuals with Duchenne muscular dystrophy. METHODS: Articles published from 1984 to 2017 were reviewed. Six articles met the inclusion criteria that included within-subject control or between-subject control group, participants with a diagnosis of only Duchenne muscular dystrophy, participation in respiratory muscle training intervention, and outcome measures of endurance and strength. Effect sizes were calculated for each study and overall, weighted mean effect sizes for strength and endurance outcome measures. RESULTS: There was a large effect for improving respiratory endurance and a moderate effect for muscle strength. However, these effects were not significant. CONCLUSION: Findings justify further exploration of the potential benefits of respiratory muscle training for individuals with Duchenne muscular dystrophy.

European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials.

Development of novel therapies for Duchenne muscular dystrophy (DMD) are driving the need for more efficient ways of detecting changes in disease- progression in DMD [1]. However, medicines' approval must be based on outcome measures that are acceptable from a regulatory perspective. In this article, European regulators provide an update on the recent regulatory consideration of a new endpoint (Stride Velocity 95th Centile (SV95C)) that could be used in therapeutic DMD trials. This new endpoint aims to quantify a patient's ambulation directly, reliably and continuously in a home environment with a wearable device.