U.S., July 9 -- ClinicalTrials.gov registry received information related to the study (NCT07052929) titled 'Study of ASP2957 in Male Patients With X-linked Myotubular Myopathy Who Need Ventilators' on June 30.
Brief Summary: X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no therapies for XLMTM.
The protein myotubularin is needed for muscle development and movement. A gene called MTM1 tells the body to make myotubularin. XLMTM is caused by changes, or mutations, in the MTM1 gene. Changes in the MTM1 gene causes low or no levels of myotubularin to be made, so the muscles do not work properly.
Gene therapy is a way of getting a healthy copy of...