Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for early intervention.
Clinical Brief: Natural Progression and Assessment Challenges in SMA
Main Discussion Topics
Key Points for Physicians
Notable Insights
There is a significant disconnect between measurable outcomes on standardized scales and meaningful functional improvements that matter to patients in their daily lives, creating challenges for treatment authorization.
Clinical Significance
Physicians must advocate for treatment based on combined standardized assessments and patient-reported outcomes, recognizing that existing scales may not adequately capture clinically meaningful changes in older patients with SMA.
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