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Randomized Controlled Trial
. 2025 Sep 11;272(9):626.
doi: 10.1007/s00415-025-13362-z.

Efficacy and safety of respiratory strength and endurance training in patients with myotonic dystrophy type 1 (DM1): a randomized controlled trial

Stephan Wenninger  1 , Eva Heidsieck  2 , Corinna Wirner-Piotrowski  2 , Marko Mijic  2 , Natalia Garcia-Angarita  2 , Kristina Gutschmidt  2 , Daniel H Mendelshohn  2 , Benedikt Schoser  2
Affiliations
Randomized Controlled Trial

Efficacy and safety of respiratory strength and endurance training in patients with myotonic dystrophy type 1 (DM1): a randomized controlled trial

Stephan Wenninger et al. J Neurol. .

Abstract

Background: Myotonic dystrophy type 1 (DM1) is a multisystem disorder characterized by progressive muscle weakness, including the respiratory muscles, which often leads to ventilatory insufficiency. Despite its clinical relevance, high-quality controlled data on the effects of respiratory muscle training in DM1 are limited.

Objective: To evaluate the safety and effectiveness of long-term, home-based inspiratory muscle training (IMT) using a commercially available inspiratory training device in genetically confirmed DM1 patients.

Methods: This was a single-center, randomized controlled trial involving three parallel groups: inspiratory strength training, inspiratory endurance training, and a control group without training. Patients were followed over 9 months. The primary outcome was the change in maximal inspiratory pressure (MIP) after nine months. Secondary outcomes included forced vital capacity, maximal expiratory pressure, capillary blood gases, a 6-min walk test, and validated patient-reported outcomes. Adherence and safety were monitored.

Results: Twenty-six participants completed the study. The intervention was safe, with no serious adverse events. Significant improvements in primary outcome MIP were observed in both intervention groups compared to control (p < 0.05), with the strength group showing the greatest benefit. Secondary outcomes improved significantly in the endurance group. Adherence to the training exceeded 80% across all groups. Baseline differences in MIP, FVC, and FEV1 were observed and considered in the analysis.

Conclusion: Regular IMT is a feasible, safe, and effective intervention for improving respiratory function in patients with DM1. Regular RMT can enhance respiratory muscle strength and endurance and should be incorporated into the multidisciplinary care of DM1 patients showing initial signs of restrictive ventilatory insufficiency.

Keywords: DM1: Myotonic Dystrophy Type 1; Endurance training; IMT: Inspiratory mucle training; Respiratory muscle training.

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Conflict of interest statement

Declarations. Conflicts of interest: All authors report no disclosures regarding this study. Outside of this context, SW has received research grant from the DGM – Deutsche Gesellschaft für Muskelkranke e.V. He has served on advisory boards for Alexion Pharma, CSL Behring, and Sanofi Genzyme GmbH. He received funding for travel or speaker honoraria from Sanofi-Aventis Germany GmbH; SH Glykogenose Gesellschaft; AbbVie Germany GmbH; Recordati Pharma GmbH; CSL Behring GmbH; Alexion Pharma GmbH; Desitin Germany; Akcea GmbH. Outside of this context, BS has served on advisory boards for Alexion, Argenex, Amicus, Astellas, Spark, and Sanofi; he has undertaken contracted unrestricted research for Sanofi and Amicus; and has received honoraria from Kedrion. The other authors have no conflicts of interest to declare. Ethical approval: The study was approved by the ethics committee of the LMU Klinikum, Project No. 19-330, and the protocol was registered on a public clinical trials registry (ClinicalTrials.gov Identifier NCT04052958).

Figures

Fig. 1
Fig. 1
Boxplot change in MIP from BL (Month 0, M0) to EOS (Month 9, M9)
Fig. 2
Fig. 2
Change in MIP from BL (Month 0, M0) to EOS (Month 9, M9). Note: due to the restrictions during the COVID-19 pandemic, only a total of 10 participants attended at visit month 5, and only one of them was a patient from the control group
See this image and copyright information in PMC

References

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