NMD, neuromuscular disease, neuromuscular disorder, muscle disease, Duchenne muscular dystrophy, DMD, congenital myopathy, CM, myotonic dystrophy type 1, MD1, DM1, spinal muscular atrophy, SMA, limb girdle muscular dystrophy, LGMD, congenital muscular dystrophy, CMD Muscle disease
Conditions
Interventions
The study spans 24 weeks, consisting of two phases of each twelve weeks. The participants will perform supervised training, unsupervised training, and/or no training. Training consists of five inspira
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
2 Years to 64 Years
Inclusion criteria
Inclusion criteria: Proven NMDYounger than 18 years old at the first study visit. Able to participate and comply with study requirements. If > 12 years: willingness and ability to understand nature and content of the study If < 16 years: legal representative with willingness and ability to understand nature and content of the study Able to perform spirometry and respiratory muscle strength testing.
Exclusion criteria
Exclusion criteria: History of pneumothorax in the past 6 weeks Known heart failure (EF < 40%). History of other conditions that affect pulmonary or respiratory muscle function. Pneumonia in past six weeks. Patients who have recently (<2 months) started medication treatment and/or gene therapy that could potentially affect respiratory status. Scoliosis surgery in the past 4 months. Patient and legal representative unable to speak and understand Dutch or English. Respiratory muscle training (loaded) in the past 3 months. > 22/24 hours non-invasive ventilation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Efficacy: change in MIP from baseline to twelve weeks after initiation of RMT. | — |
Secondary
| Measure | Time frame |
|---|---|
| Feasibility:Adherence: completion rate of the number of training sessions Enjoyment of the training with a small questionnaire: every two weeks during RMTIntensity of the trainingFrequency of the trainingQuestionnaire‘Algehele ervaring’ after 24 weeks of RMT Efficacy of twelve and 24 weeks of RMT:Questionnaires‘Algehele ervaring’ after 24 weeks of RMT‘Dagelijks functioneren’ at baseline, after twelve weeks of RMT, and after 24 weeks of RMTMIP, MEP, SNIP, peak expiratory cough flow (PECF), vital capacity (VC), FVC, FEV1, and peak expiratory flow (PEF) measured at baseline, after twelve weeks of RMT, and after 24 weeks of RMT Efficacy of supervision:All parameters/endpoints as mentioned above will be used. | — |
Countries
Netherlands
Contacts
Public ContactE.S. Veldhoen
Universitair Medisch Centrum Utrecht
Outcome results
None listed