Journal of neuromuscular diseases
New SMA era: A broad-range tiered assessment of function for the evolving SMA phenotype (EVOLVE-SMA).
Meghan Moore Burk, Thomas O Crawford, Kristen Johnson, Susan Apkon, Laurey Brown, Terri Carry, Katie Hoffman, Carolyn Kelley, Nancy L Kuntz, Julie Miller, Jessica Nance, Ruchee Patel, Vamshi Rao, Carly Rothman, Abigail Schwaede
Published: 202510.1177/22143602251405346
Abstract
BACKGROUND: The clinical course of Spinal Muscular Atrophy (SMA) has been transformed by new disease-modifying therapies (DMT). Before DMT, the clinical course of SMA was marked by an inflection point between developmental improvement and degenerativ…
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