Clinical Trial: A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957

U.S., Oct. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07876804) titled 'A Long-term Follow-up Study in Male Participants With X-linked Myotubular Myopathy Treated With ASP2957' on Oct. 09.

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 approved therapies for XLMTM.

The protein myotubularin is needed for muscle development, movement and breathing. 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 cause low or no levels of myotubularin to be made, so the muscles do not work properly.

Gene therapy is...