X-linked Myotubular Myopathy Clinical Trial
Official title:
Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
This study is a longitudinal study evaluating the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14.
Subjects aged 0-7 will be evaluated every six months for a total of three evaluations (Baseline, Six-Month Visit, Twelve-Month Visit). Subjects aged 8-14 will be evaluated every twelve months for a total of two evaluations (Baseline, Twelve-Month Visit). Evaluations include medical history, physical exam, respiratory muscle tests, a qualitative interview related to the child's function and use of respiratory aids, and quality of life assessments. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
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| Completed |
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A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects
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| Active, not recruiting |
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Gene Transfer Clinical Study in X-Linked Myotubular Myopathy
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Phase 2/Phase 3 | |
| Completed |
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N/A |