scholarly journals Effects of yoga breathing exercises on pulmonary function in patients with Duchenne muscular dystrophy: an exploratory analysis

2014 ◽  
Vol 40 (2) ◽  
pp. 128-133 ◽  
Author(s):  
Marcos Rojo Rodrigues ◽  
Celso Ricardo Fernandes Carvalho ◽  
Danilo Forghieri Santaella ◽  
Geraldo Lorenzi-Filho ◽  
Suely Kazue Nagahashi Marie
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Pulmonary Function ◽  
Respiratory Function ◽  
University Hospital ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Study ◽  
Breathing Exercises ◽  
The City

OBJECTIVE: Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in children, and children with DMD die prematurely because of respiratory failure. We sought to determine the efficacy and safety of yoga breathing exercises, as well as the effects of those exercises on respiratory function, in such children. METHODS: This was a prospective open-label study of patients with a confirmed diagnosis of DMD, recruited from among those followed at the neurology outpatient clinic of a university hospital in the city of São Paulo, Brazil. Participants were taught how to perform hatha yoga breathing exercises and were instructed to perform the exercises three times a day for 10 months. RESULTS: Of the 76 patients who entered the study, 35 dropped out and 15 were unable to perform the breathing exercises, 26 having therefore completed the study (mean age, 9.5 ± 2.3 years; body mass index, 18.2 ± 3.8 kg/m2). The yoga breathing exercises resulted in a significant increase in FVC (% of predicted: 82.3 ± 18.6% at baseline vs. 90.3 ± 22.5% at 10 months later; p = 0.02) and FEV1 (% of predicted: 83.8 ± 16.6% at baseline vs. 90.1 ± 17.4% at 10 months later; p = 0.04). CONCLUSIONS: Yoga breathing exercises can improve pulmonary function in patients with DMD.

scholarly journals Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

PLoS Medicine ◽  
2020 ◽  
Vol 17 (9) ◽  
pp. e1003222 ◽  
Author(s):  
Edward C. Smith ◽  
Laurie S. Conklin ◽  
Eric P. Hoffman ◽  
Paula R. Clemens ◽  
Jean K. Mah ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Interim Analysis ◽  
Extension Study ◽  
Efficacy And Safety ◽  
Open Label ◽  
Open Label Extension

The effect of trunk training on trunk control, upper extremity, and pulmonary function in children with Duchenne muscular dystrophy: A randomized clinical trial

2021 ◽  
pp. 026921552110432
Author(s):  
Gökçe Yağmur Güneş Gencer ◽  
Öznur Yilmaz
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Pulmonary Function ◽  
Respiratory Function ◽  
Exercise Program ◽  
Control Group ◽  
Study Group ◽  
Trunk Control ◽  
Intergroup Comparison ◽  
Arm Function

Objectives: To investigate the effect of trunk training on trunk control, arm, and pulmonary function in children with Duchenne muscular dystrophy. Design: A randomised controlled trial. Settings: Neuromuscular diseases clinic of university hospital. Subjects: Twenty-six children with Duchenne muscular dystrophy aged 5–16 were included in the study. Intervention: Participants were randomly allocated into two groups. The study group ( N = 13) exercised with the trunk-oriented exercise program and the conventional exercise program, whereas the control group ( N = 13) underwent the conventional exercise program for eight weeks. Main measures: The primary outcomes were trunk control was assessed using the Trunk Control Measurement Scale, the arm function was assessed using Performance of Upper Limb, and respiratory function using the pulmonary function test. Data collection was conducted at baseline, and eighth week. The differences in trunk control scores, arm function scores, and respiratory function values before and after the training were calculated for the intergroup comparison. Results: The mean age of the participants was 11.6 (2.6) in the study group and 10.6 (3.4) in the control group. The changes between trunk control score, arm function score (total and distal level score), and respiratory function value (Forced Vital Capacity, Forced Expiratory Volume in one second, and Peak Expiratory Flow Volume percentage values) were compared and significant differences were found after eight week periods in the study and control groups. Conclusions: Trunk-oriented exercise program in Duchenne muscular dystrophy might be effective for trunk control, arm, and respiratory function.

scholarly journals Perioperative Evaluation of Respiratory Muscle Strength after Scoliosis Correction in Patients with Duchenne Muscular Dystrophy

2017 ◽  
Vol 11 (5) ◽  
pp. 787-792 ◽  
Author(s):  
Wataru Saito ◽  
Kosuke Mizuno ◽  
Gen Inoue ◽  
Takayuki Imura ◽  
Toshiyuki Nakazawa ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Pulmonary Function ◽  
Respiratory Function ◽  
Respiratory Muscle ◽  
Vital Capacity ◽  
Respiratory Muscle Strength ◽  
Scoliosis Correction ◽  
The Mean

<sec><title>Study Design</title><p>Retrospective cohort study.</p></sec><sec><title>Purpose</title><p>To investigate the effect of spinal correction on respiratory muscle strength in patients with Duchenne muscular dystrophy (DMD).</p></sec><sec><title>Overview of Literature</title><p>Several studies have reported that scoliosis correction in patients with DMD does not improve pulmonary function. In these studies, pulmonary function was evaluated using the traditional spirometric values of percent vital capacity (%VC) and percent forced vital capacity (%FVC). However, traditional spirometry may not be suitable for patients with DMD because the results can be influenced by patient fatigue or level of understanding. Therefore, we evaluated respiratory function focusing on respiratory muscle strength using maximal inspiratory pressure (MIP), maximal expiratory pressure (MEP), and sniff nasal inspiratory pressure (SNIP), in addition to %VC and %FVC.</p></sec><sec><title>Methods</title><p>We retrospectively reviewed 16 patients with DMD who underwent spinal correction surgery between 2006 and 2011 at Kitasato University Hospital. All patients were males, and the mean age was 13.5 years. Respiratory muscle strength was evaluated using MIP, MEP, and SNIP. Measurements were obtained preoperatively and at 1 and 6 months postoperatively, and %VC and %FVC were obtained preoperatively and within 6 months postoperatively.</p></sec><sec><title>Results</title><p>The mean preoperative and postoperative %VC values were 54.0% and 51.7%, whereas the mean %FVC values were 53.9% and 53.2%, respectively. The mean MIP, MEP, and SNIP values obtained preoperatively and at 1 and 6 months postoperatively were as follows: MIP, 40.5, 42.7 and 47.2 cm H<sub>2</sub>O; MEP, 26.0, 28.0, and 29.0 cm H<sub>2</sub>O; and SNIP, 33.4, 33.0, and 33.0 cm H<sub>2</sub>O; respectively. The mean MIP and MEP values significantly improved postoperatively. There were no significant differences in SNIP, %VC, or %FVC preand postoperatively.</p></sec><sec><title>Conclusions</title><p>By focusing on respiratory muscle strength, our results suggest that scoliosis correction in patients with DMD might have a favorable effect on respiratory function.</p></sec>

scholarly journals Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

2021 ◽  
pp. 1-13
Author(s):  
Craig M. McDonald ◽  
Perry B. Shieh ◽  
Hoda Z. Abdel-Hamid ◽  
Anne M. Connolly ◽  
Emma Ciafaloni ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Adverse Events ◽  
Exon Skipping ◽  
Control Group ◽  
Phase 2 ◽  
Open Label ◽  
M Phase ◽  
Positive Treatment

Background Eteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 51 skipping, based on demonstrated dystrophin production. Objective To report results from PROMOVI, a phase 3, multicenter, open-label study evaluating efficacy and safety of eteplirsen in a larger cohort. Methods Ambulatory patients aged 7–16 years, with confirmed mutations amenable to exon 51 skipping, received eteplirsen 30 mg/kg/week intravenously for 96 weeks. An untreated cohort with DMD not amenable to exon 51 skipping was also enrolled. Results 78/79 eteplirsen-treated patients completed 96 weeks of treatment. 15/30 untreated patients completed the study; this cohort was considered an inappropriate control group because of genotype-driven differences in clinical trajectory. At Week 96, eteplirsen-treated patients showed increased exon skipping (18.7-fold) and dystrophin protein (7-fold) versus baseline. Post-hoc comparisons with patients from eteplirsen phase 2 studies (4658-201/202) and mutation-matched external natural history controls confirmed previous results, suggesting clinically notable attenuation of decline on the 6-minute walk test over 96 weeks (PROMOVI: –68.9 m; phase 2 studies: –67.3 m; external controls: –133.8 m) and significant attenuation of percent predicted forced vital capacity annual decline (PROMOVI: –3.3%, phase 2 studies: –2.2%, external controls: –6.0%; p <  0.001). Adverse events were generally mild to moderate and unrelated to eteplirsen. Most frequent treatment-related adverse events were headache and vomiting; none led to treatment discontinuation. Conclusions This large, multicenter study contributes to the growing body of evidence for eteplirsen, confirming a positive treatment effect, favorable safety profile, and slowing of disease progression versus natural history.

P4.8 Results from a two-year open label intervention study with idebenone (Catena®) in Duchenne muscular dystrophy

2011 ◽  
Vol 21 (9-10) ◽  
pp. 706
Author(s):  
G.M. Buyse ◽  
D. Thijs ◽  
N. Goemans ◽  
M. van den Hauwe ◽  
H. Wei ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Intervention Study ◽  
Open Label
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