Chronic respiratory failure after the first dose of nusinersen treatment: A case report

Özhan Orhan(1), Mehmet Nur Talay(2), Halime Nayır Büyükşahin(3), Asuman Demirbuğa(4), Mehmet Nuri Özbek(5), Nezir Özgün(6)
(1) Mardin Artuklu University, Faculty of Medicine, Department of Pediatrics, Mardin, Türkiye,
(2) Mardin Artuklu University, Faculty of Medicine, Department of Pediatrics, Mardin, Türkiye,
(3) Mardin Training and Research Hospital, Department of Pediatric Pulmonology, Mardin, Türkiye,
(4) Mardin Training and Research Hospital, Department of Pediatric Infection, Mardin, Türkiye,
(5) Mardin Artuklu University, Faculty of Medicine, Department of Pediatric Endocrinology, Mardin, Türkiye,
(6) Mardin Artuklu University, Faculty of Medicine, Department of Pediatric Neurology, Mardin, Türkiye

Abstract

Spinal muscular atrophy (SMA) is a disease characterized by progressive nerve cell loss in the spinal cord and brainstem. It can be fatal if left untreated, particularly in infantile forms. Nusinersen became the first disease-modifying agent to be approved in 2016 for the treatment of all types of 5q-associated SMA, across all age groups. Trials have shown that the drug has an acceptable safety profile and has significantly improved clinical care for patients; however, data on long-term risks and benefits are limited. Respiratory problems following use of the drug are an important potential complication and the mechanisms of action are not fully understood. This study reports on a patient with type 1 SMA who developed chronic respiratory failure after the first dose of nusinersen, a condition not previously reported. Although nusinersen significantly improves patients' quality of life, its long-term or rare side effects are not well understood. Patients should be closely monitored for respiratory side effects, especially after the first dose, and families should be thoroughly informed about potential respiratory complications.


 


How to cite this article:


Orhan Ö, Talay MN, Büyükşahin HN, Demirbuğa A, Özbek MN, Özgün N. Chronic respiratory failure after the first dose of nusinersen treatment: A case report. J Med Dent Invest 2024;5:e240341. https://doi.org/10.5577/jomdi.e240341

Full text article

Generated from XML file

Authors

Özhan Orhan
ozhan.orhan@hotmail.com.tr (Primary Contact)
Mehmet Nur Talay
Halime Nayır Büyükşahin
Asuman Demirbuğa
Mehmet Nuri Özbek
Nezir Özgün
1.
Orhan Özhan, Talay MN, Nayır Büyükşahin H, Demirbuğa A, Özbek MN, Özgün N. Chronic respiratory failure after the first dose of nusinersen treatment: A case report. J Med Dent Invest. 2024;5:e240341. doi:10.5577/jomdi.e240341

Article Details

How to Cite

1.
Orhan Özhan, Talay MN, Nayır Büyükşahin H, Demirbuğa A, Özbek MN, Özgün N. Chronic respiratory failure after the first dose of nusinersen treatment: A case report. J Med Dent Invest. 2024;5:e240341. doi:10.5577/jomdi.e240341
Smart Citations via scite_

Similar Articles

You may also start an advanced similarity search for this article.

No Related Submission Found