Clinical trials: X-linked Myotubular Myopathy

X-Linked Myotubular Myopathy

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

• Active, not recruiting
• Age: Up to 5 years
• Phase 2/3
• Injection or infusion
• Results posted

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 this serious condition.

Trial name

ASPIRO (NCT03199469)

Testing

Resamirigene bilparvovec

Where

6 sites in United States, Canada and 2 more

Volunteers

27 people

Sponsor

Astellas Gene Therapies