A Real-World Survey of Spinal Muscular Atrophy: Uncovering the Persistent Burden in Treated Patients

Despite the availability of approved SMN-targeted therapies, patients living with spinal muscular atrophy (SMA) continue to face a significant and persistent disease burden. That’s why Precision AQ’s Health Economics and Research Outcomes (HEOR) team developed two complementary, web-based surveys across adult patients and caregiver proxies of pediatric patients to generate rigorous, peer-reviewed real-world evidence to support the next-generation SMA therapy.

 

What You'll Learn:

• The extent of patient and caregiver burden and its impact on quality of life, well-being, and productivity
• Ways validated patient-reported outcome measures, including PROMIS Fatigue SF and EQ-5D-5L, were implemented to capture clinical, humanistic, and productivity burden
• How Precision AQ translated lived patient experience into evidence that strengthens value narratives and informs reimbursement strategies

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Topics:   HEOR & RWE