The long-term efficacy of nusinersen on respiratory functions in children with symptomatic spinal muscular atrophy type 1
Improvement in respiratory function in patients treated with nusinersen for 2 years or more is generally not expected in the natural course of the disease. However, in our cohort, patients treated with nusinersen mainly maintained their current respiratory status, and many patients required ventilatory support despite treatment with nusinersen. Therefore, our findings may reflect the limited efficacy of nusinersen in symptomatic patients.
Authors
- Hülya Maraş Genç (ORCID: https://orcid.org/0000-0001-8869-0277)
- Fulya Kürekçi (ORCID: https://orcid.org/0000-0002-6872-8781)
- Mehmet Akif Kılıç (ORCID: https://orcid.org/0000-0002-6571-8449)
- Selahattin Katar (ORCID: https://orcid.org/0000-0002-5195-2832)
- Erkan Çakır (ORCID: https://orcid.org/0000-0002-1438-7854)
- Osman Kipoğlu (ORCID: https://orcid.org/0000-0001-9208-6697)
- Rıdvan Avci (ORCID: https://orcid.org/0000-0002-8210-1280)
- Burçin Nazlı Karacabey (ORCID: https://orcid.org/0000-0001-8857-4356)
- Edibe Pembegül Yıldız (ORCID: https://orcid.org/0000-0002-8016-0404)
- Orhan Coşkun (ORCID: https://orcid.org/0000-0001-9229-404X)
Institutions
- Istinye University (TR)
- Istanbul University (TR)
Publication Details
- Journal
- TURKISH JOURNAL OF MEDICAL SCIENCES
- Published
- 2025-06-20
- DOI
- https://doi.org/10.55730/1300-0144.6018
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00