A UK modified Delphi consensus from 19 experts produced 22 statements recommending that patients with spinal muscular atrophy (SMA) on permanent ventilation should not be excluded from disease-modifying therapies. The panel called for broader outcome measurement using functional, respiratory, and quality-of-life endpoints rather than narrower criteria. The guidance responds to a common real-world access issue in which ventilated patients may be treated as outside eligibility boundaries due to historical endpoints or pragmatic trial limitations. Consensus statements can shape payer decisions and clinician interpretation even before new regulatory labels or trial results arrive. For the SMA community, the immediate impact is clarity on how to evaluate benefit where traditional endpoints may underrepresent clinically meaningful respiratory and functional outcomes.
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