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Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment (COMPIS)
Congenital myopathies (CM) is a large group of muscle disorders, presenting with hypotonia and non-progressive generalised muscle weakness, which can lead to motor developmental delay.More than 20 genes can cause CM and currently there is no curative treatment for this disorder. Case reports and a smaller study have previous reported that oral salbutamol has benefited subjects with different types of congenital myopathies by increasing their muscle strength.The exact effect of salbutamol in muscle cells isn't exactly known but it has been hypothesized to have an anabolic effect by triggering different pathways inside the muscle cells which increase cell proliferation, decrease apoptosis, decreases proteolysis and increases protein synthesis. The aim of our study is evaluate if daily oral salbutamol can increase the muscle function and muscle strength in these patients after 6 months on treatment, compared to no treatment.
Details
| Lead sponsor | Vastra Gotaland Region |
|---|---|
| Phase | NA |
| Status | COMPLETED |
| Enrolment | 18 |
| Start date | Mon Oct 25 2021 00:00:00 GMT+0000 (Coordinated Universal Time) |
| Completion | Mon Mar 17 2025 00:00:00 GMT+0000 (Coordinated Universal Time) |
Conditions
- Congenital Myopathy
- Neuromuscular Diseases
- Musculoskeletal Diseases
- Nemaline Myopathy
- Centronuclear Myopathy
- Myosin Storage Myopathy
Interventions
- Salbutamol (as Salbutamol Sulfate) 2 Mg Oral Tablet
- Salbutamol Only Product in Oral Dose Form
Countries
Sweden