September 2026

NEUROSCIENCES

ADULTS WITH SPINAL MUSCULAR ATROPHY FACE NEW CHALLENGES IN THE TREATMENT ERA

In a recent NeurologyLive discussion, Senda Ajroud-Driss, MD, director of the Lois Insolia ALS Clinic and Muscular Dystrophy Association Clinic at Northwestern Medicine, highlighted the evolving needs of adults living with spinal muscular atrophy (SMA) in an era of rapidly advancing treatments.

Key Takeaways
  • Disease-modifying therapies have improved outcomes for many patients with spinal muscular atrophy (SMA), but adults continue to have significant unmet needs.
  • Respiratory support, nutritional management, rehabilitation and swallowing evaluation remain critical components of care.
  • Current outcome measures may not adequately capture meaningful treatment benefits experienced by adults with SMA, including changes in fatigue, strength and function.
  • Questions remain about treatment monitoring, switching therapies and the role of combination approaches in adult populations
  • Access to specialized multidisciplinary care and delays in diagnosis continue to create challenges for patients.
 
New Therapies Have Changed SMA Care
Disease-modifying therapies have transformed care for patients with SMA, improving outcomes and reducing serious complications. According to Senda Ajroud-Driss, MD, many adults also experience improvements in fatigue and muscle strength. However, adults with longstanding weakness continue to face challenges that require ongoing support beyond medication alone.

Why Multidisciplinary Care Still Matters
Dr. Ajroud-Driss emphasized that respiratory care, nutritional support, rehabilitation services and swallowing evaluations remain essential components of adult SMA care. While therapies may stabilize disease progression, they do not eliminate the need for coordinated care aimed at preserving function and independence.

In Northwestern Medicine's adult multidisciplinary clinic, care extends beyond neurologic management to include pulmonology, nutrition, rehabilitation and speech-language pathology.

Evidence Gaps in Adult SMA
Much of the evidence supporting today's SMA therapies comes from pediatric studies. As a result, clinicians continue to face unanswered questions about how best to assess treatment response in adults, when therapies should be changed and whether combination approaches may provide additional benefit. Current outcome measures may not fully capture meaningful improvements in adults with SMA.

For adults with SMA, the future is promising. Continued research focused on adult populations, improved assessment tools and integrated care models may help close existing gaps and further enhance outcomes for patients benefiting from a rapidly evolving therapeutic landscape.
Senda Ajroud-Driss, MD, Director of the Lois Insolia ALS Clinic and Muscular Dystrophy Association Clinic and Professor of Neurology at Northwestern Medicine

Refer a Patient

Northwestern Medicine welcomes the opportunity to partner with you in caring for your patients. ​

You May Also Like