Musculoskeletal Diseases
Conditions
Interventions
No pyridostigmine
Sponsors
University College London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Children between the ages of 0-18 years Molecular diagnosis of centronuclear myopathy Evidence of decremental activity of greater than 10% on repetitive nerve stimulation testing
Exclusion criteria
Exclusion criteria: Absence of a genetically confirmed diagnosis of centronuclear myopathy Absence of decremental activity on repetitive nerve stimulation testing
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Response to the medication in terms of muscle strength, as graded by Medical Research Council (MRC) scale, distance covered in the 6 minute walk test (6MWT) for ambulant patients and the impact of drug trial on activities of daily living (ADL’s). | — |
Secondary
| Measure | Time frame |
|---|---|
| The number of admissions to hospital. | — |
Countries
South Africa
Contacts
Public ContactSharika Raga
PhD fellow in paediatric neuromuscular diseases
Outcome results
None listed