Opinion
Video
Assessing Different Factors When Selecting a Treatment
Author(s):
Panelists discuss how comorbidities such as scoliosis, dislocated hips, and nutritional issues affect spinal muscular atrophy (SMA) treatment decisions, with treatment approaches evolving as patients' functional abilities improve with disease-modifying therapies.
Clinical Brief: Comorbidities and Changing Phenotypes in Patients With Treated SMA
Main Discussion Topics
- Impact of comorbidities on treatment decisions and management
- Challenges in addressing orthopedic issues in functionally improving patients
- Evolving patterns of scoliosis and spinal deformities in treated patients
- Reproductive considerations for patients with SMA
Key Points for Physicians
- Treatment has changed the natural history of SMA, creating new clinical challenges in managing comorbidities.
- Orthopedic interventions (for dislocated hips, scoliosis) require reassessment as patients gain function.
- Kyphosis is becoming more prominent than typical scoliosis in treated patients.
- Genetic counseling remains essential as disease-modifying therapies do not alter genetic status.
Notable Insights
The panel noted that disease-modifying treatments are not curative and do not change genetic inheritance patterns, necessitating continued genetic counseling for family planning.
Clinical Significance
As disease-modifying therapies alter the natural history of SMA, physicians must reconsider traditional approaches to comorbidities and develop new management paradigms for this evolving clinical landscape.
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