Trial ID or NCT#





This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe. The duration of the study, including the enrollment period, will be 36 months. Data from the study will be used to characterize the disease course of MTM and determine which outcome measures will be the best to assess the efficacy of potential therapies.

Official Title

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

Eligibility Criteria

Sexes Eligible for Study: All
Accepts Healthy Volunteers: No


Casey H. Halpern, MD
Functional neurosurgeon
Assistant Professor of Neurosurgery and, by courtesy, of Neurology and of Psychiatry and Behavioral Sciences at the Stanford University Medical Center
Sarada Sakamuri, MD
Neuromuscular neurologist, General neurologist, Neurophysiologist
Clinical Assistant Professor, Neurology & Neurological Sciences

Contact us to find out if this trial is right for you.


Spectrum Child Health
(650) 724-1175