Bridging the Gap: Addressing Unmet Needs in Spinal Muscular Atrophy for Older Patients 

home / peer-exchange / bridging-the-gap-addressing-unmet-needs-in-spinal-muscular-atrophy-for-older-patients

Julie A. Parsons, MD; John Brandsema, MD; and Tina Duong, MPT, PhD, discuss how management approaches for older patients with spinal muscular atrophy (SMA) differ from those for infants, highlighting the importance of individualized treatment goals focused on independence, fatigue management, quality of life, and the challenges of measuring outcomes with currently available assessment tools, while also exploring current and emerging therapies, including splice modifiers and potential intrathecal gene therapy options.

Episodes

May 15th 2025EP. 1: Describing SMA Progression Over Time

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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).

May 15th 2025EP. 2: Unmet Needs of Older Patients Living With SMA

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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.

May 22nd 2025EP. 3: Examining the Importance of Early Treatment for SMA

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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

May 22nd 2025EP. 4: Key Functional Milestones Affected by Disease Progression

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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

May 29th 2025EP. 5: The Importance of Supportive Care for Patients Living With SMA

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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.

May 29th 2025EP. 6: Introduction to Therapies Used to Treat SMA

Julie Parsons, MD;John Brandsema, MD;Tina Duong, PhD, MPT

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.