Completed | cmdir.org

The study has ended normally, and participants are no longer being examined or treated (that is, the last participant's last visit has occurred).

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

A Rollover Study of ARRY-371797 in Patients With LMNA-Related Dilated Cardiomyopathy

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

Congenital Muscle Disease Hypoglycemia Survey

MTM Manifesting Carrier Study

Myotubular Myopathy Event Study (MTMES)

Myotubular Myopathy Genetic Testing Study

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)

Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)

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