| Description |
Traditionally, infants with spinal muscular atrophy Type I (SMA1) demonstrated poor functional outcomes and did not survive past infancy. New disease-modifying therapies have shown initial success in improving functional outcomes for these children and directly impacts the current standard of care for children with SMA1. PTs will inevitably treat patients with SMA1 receiving these innovative pharmaceutical interventions and should be aware of the unique clinical presentation when compared to children that have not received this medication. The novelty of these therapies changes what pediatric PTs need to know about children with SMA1, creating a gap in the literature regarding clinical presentation, interventions, and functional outcomes. These kinds of complex clinical cases require clinicians to demonstrate adaptive expertise and the ability to learn and innovate in response to practice challenges. This session will discuss the clinical presentation of 5 children with SMA1 receiving Spinraza highlighting the patterns of movement for this patient population and the functional outcomes achieved. The PT’s demonstration of adaptive expertise and progressive problem solving that underlies this complex clinical reasoning process will be emphasized. The impact of SMA 1 on the neuromuscular and cardiovascular and pulmonary systems will be reviewed with integration of supporting evidence for intervention.
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