A functional composite measure of spinal and bulbar muscular atrophy

Takeaway

  • Spinal and bulbar muscular atrophy functional composite (SBMAFC) is an efficient and sensitive tool for detecting early-stage spinal and bulbar muscular atrophy (SBMA) and monitoring disease progression.

Why this matters

  • The potential goal of disease-modifying drugs in the treatment of SBMA is to slow down progression. A reliable and objective measurement for muscular strength and function is an unmet need.

  • This functional composite can be an outcome measure in future clinical trials of therapies targeting SBMA.