Clinical outcome measures in congenital muscular dystrophy

Takeaway

  • The Motor Function Measure 32 (MFM32) is a sensitive outcome measure in children with COL6-related dystrophies (COL6-RD) and LAMA2-related dystrophies (LAMA2-RD).

Why this matters

    The MSMF32 should be used as an outcome measure in future clinical trials to evaluate disease progression in children with congenital muscular dystrophy (CMD).