Myotubular Myopathy Genetic Testing Study

CompletedOBSERVATIONAL
Enrollment

23

Participants

Timeline

Start Date

March 31, 2013

Primary Completion Date

March 31, 2017

Study Completion Date

March 31, 2017

Conditions
Myotubular Myopathy
Interventions
OTHER

Genetic Testing

Genetic testing services will be provided by the University of Chicago under the guidance of Dr. Soma Das, PhD. Services will be coordinated by Rachel Alvarez, CMDIR Associate Director. Genetic testing will include standard sequencing of the MTM1 gene followed by CGH array for deletion/duplication in cases where no pathologic variant is identified by standard sequencing.

Trial Locations (1)

90502

Congenital Muscle Disease International Registry, Torrance

All Listed Sponsors
collaborator

Valerion Therapeutics, LLC

INDUSTRY

collaborator

Congenital Muscle Disease International Registr

OTHER

collaborator

University of Chicago

OTHER

collaborator

Boston Children's Hospital

OTHER

lead

Cure CMD

OTHER

NCT01817946 - Myotubular Myopathy Genetic Testing Study | Biotech Hunter | Biotech Hunter