Julie Parsons, MD

Panelists discuss how disease progression despite treatment in older patients with spinal muscular atrophy (SMA) may be due to natural aging effects combined with SMA, not necessarily treatment failure, highlighting the importance of exercise, nutrition, and management of contractures.

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered intrathecally quarterly, both showing similar safety and efficacy profiles.

Panelists discuss how the current spinal muscular atrophy (SMA) treatment landscape includes 3 options: gene therapy (onasemnogene abeparvovec [Zolgensma]) for younger patients and 2 splice modifiers (nusinersen [Spinraza] and risdiplam [Evrysdi]) for older patients.

Panelists discuss how supportive care for patients with spinal muscular atrophy (SMA) includes rehabilitation, respiratory care, psychosocial support, and multidisciplinary approaches to help patients achieve independence and improved quality of life.

Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for early intervention.

Early intervention in Spinal Muscular Atrophy is crucial for better outcomes, emphasizing the need for timely treatment to prevent irreversible disability.

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).

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients aged 2 and older without early treatment.

FDA-approved treatments for spinal muscular atrophy differ in their mechanisms of action, safety profiles, and administration challenges, with key selection factors like patient age and gene copy number, according to Julie Parsons, MD, a professor and neurologist from the University of Colorado School of Medicine.