Treatment for Facioscapulohumeral Muscular Dystrophy (FSHD)

Evidence Base for Pharmacological Interventions

• The evidence base for FSHD does not support pharmacological intervention, unlike Duchenne muscular dystrophy where glucocorticoids have proven mortality and morbidity benefits, as stated by The Lancet Neurology 1
• The American Academy of Neurology and other guideline societies may not recommend pharmacological treatment for FSHD due to the lack of evidence, with high-quality randomized controlled trials showing no significant benefit, as referenced by 2

Supportive Management Strategies

Multidisciplinary Monitoring Schedule

• Annual comprehensive assessments are recommended by medical guidelines, including functional evaluation, physical therapy assessment, and psychosocial evaluation, although the specific details of these assessments are not fully cited, the importance of regular monitoring is emphasized 2