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<p>In recent years, three disease-modifying therapies have been approved and fully reimbursed in Poland since September 2023. Those therapies have significantly improved the disease outcome but led to new uncertainties. Doubts mainly concern older patients who have already developed complications occurring because of a lack of previous access to therapy. Joint contractures or trunk deformation are the most noticeable changes that should be examined. This work examines functional and structural changes in SMA sitter patients between the ages of 5 and 20&#xa0;years during the 12-month follow-up. Twenty-one SMA patients were divided depending on the drug program in which they participated. The study protocol included functional and structural assessments. Most of the subjects experienced an improvement in cervical rotation parameter and increased contracture of the hip and knee joints. There are differences between the patients’ body sides. Neither the contractures nor their gradual decrease or increase occur symmetrically. We conclude that all patients under study improved their motor function after 12&#xa0;months. In both groups, more significant improvement occurred in the assessment of the RULM scale than in the HFMSE scale. Improvement in function was not dependent on improvement in structural parameters.</p>

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Functional and structural analysis of SITTER patients with spinal muscular atrophy

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年3月28日

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  • Aleksandra Bieniaszewska,
  • Magdalena Sobieska,
  • Ewa Gajewska

摘要

In recent years, three disease-modifying therapies have been approved and fully reimbursed in Poland since September 2023. Those therapies have significantly improved the disease outcome but led to new uncertainties. Doubts mainly concern older patients who have already developed complications occurring because of a lack of previous access to therapy. Joint contractures or trunk deformation are the most noticeable changes that should be examined. This work examines functional and structural changes in SMA sitter patients between the ages of 5 and 20 years during the 12-month follow-up. Twenty-one SMA patients were divided depending on the drug program in which they participated. The study protocol included functional and structural assessments. Most of the subjects experienced an improvement in cervical rotation parameter and increased contracture of the hip and knee joints. There are differences between the patients’ body sides. Neither the contractures nor their gradual decrease or increase occur symmetrically. We conclude that all patients under study improved their motor function after 12 months. In both groups, more significant improvement occurred in the assessment of the RULM scale than in the HFMSE scale. Improvement in function was not dependent on improvement in structural parameters.