Natural history of facioscapulohumeral dystrophy in children

Takeaway

  • Childhood facioscapulohumeral dystrophy (FSHD) has a slowly progressive but variable disease course over 2 years of follow-up.

  • Symptoms of pain and fatigue were commonly reported.

Why this matters

  • FSHD is a common, progressive muscular dystrophy disorder with significant symptom and disease course variation, particularly in those with childhood onset.

  • These data may be used to counsel patients and provide baseline measures for future clinical trials of childhood FSHD therapies.