John Brandsema, MD

Panelists discuss how the needs of older patients with spinal muscular atrophy (SMA) differ from infants, focusing on independence, education, career goals, fatigue management, and quality of life rather than just motor milestones.

Panelists discuss how spinal muscular atrophy (SMA) progresses over time, affecting motor function differently in older patients vs infants, with experts noting the shift from traditional classification (types 1 to 4) to functional categories (nonsitters, sitters, walkers).

Access to timely treatment for spinal muscular atrophy (SMA) remains a challenge, with barriers including delays in diagnosis, frequent treatment needs, and insurance coverage gaps, which impact patient care and quality of life.

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing spinal muscular atrophy (SMA).