Nusinersen for children with type I spinal muscular atrophy: 4 years’ clinical experience in Turkish cohort
Nusinersen treatment initiated before 12 months of age, especially before 3 months of age, yielded the most favorable motor outcomes in patients with SMA type 1. Early initiation is associated with improved motor milestones and reduced need for ventilatory support. However, no significant improvements were observed in the bulbar function or in patients requiring extensive respiratory support.
Authors
- Gülhıs Deda (ORCID: https://orcid.org/0000-0002-4184-6964)
- Kürşat Bora Çarman (ORCID: https://orcid.org/0000-0002-4629-1873)
- Gökmen Zararsız (ORCID: https://orcid.org/0000-0001-5801-1835)
- Özgür Duman (ORCID: https://orcid.org/0000-0002-3313-8052)
- Uluç Yiş
- Bülent Kara (ORCID: https://orcid.org/0000-0003-3780-6596)
- Deniz Yüksel (ORCID: https://orcid.org/0000-0001-8990-023X)
- Murat Gülşen (ORCID: https://orcid.org/0000-0003-4494-9536)
- Sema Saltık (ORCID: https://orcid.org/0000-0002-6749-5795)
- Mustafa Kömür (ORCID: https://orcid.org/0000-0001-6453-7323)
- Aycan Ünalp (ORCID: https://orcid.org/0000-0002-3611-5059)
- Didem Ardıçlı (ORCID: https://orcid.org/0000-0001-7054-3623)
- Gülten Öztürk
- Figen Baydan (ORCID: https://orcid.org/0000-0001-6853-6555)
- Ömer Bektaş (ORCID: https://orcid.org/0000-0002-2514-8461)
- Hüseyin Tan (ORCID: https://orcid.org/0000-0003-3331-1828)
- Ahmet Sami Güven (ORCID: https://orcid.org/0000-0002-6085-1582)
- Onur Burak Dursun (ORCID: https://orcid.org/0000-0003-2990-9851)
- Bilge Özgör (ORCID: https://orcid.org/0000-0002-6697-7629)
- Gökçen Öz-Tuncer
- Edibe Pembegül Yıldız (ORCID: https://orcid.org/0000-0002-8016-0404)
- Elif Acar Arslan (ORCID: https://orcid.org/0000-0002-3284-107X)
- Arzu Ekici (ORCID: https://orcid.org/0000-0002-0813-7189)
- Süleyman Şahin (ORCID: https://orcid.org/0000-0001-9673-5826)
- Ayşe Aysima Özçelik (ORCID: https://orcid.org/0000-0002-9567-4176)
- Ahmet Tekin (ORCID: https://orcid.org/0000-0001-5856-7833)
- Mehmet Canpolat (ORCID: https://orcid.org/0000-0002-6877-2109)
- Merve Feyza Yüksel (ORCID: https://orcid.org/0000-0001-8981-9458)
- Aynur Ayşe Karaduman
- Ercan Demir (ORCID: https://orcid.org/0009-0002-0447-7431)
- Seda Kanmaz
- Özlem Hergüler
Institutions
- Ankara University (TR)
- Karadeniz Technical University (TR)
- Dokuz Eylül University (TR)
- Akdeniz University (TR)
- Inonu University (TR)
- Ondokuz Mayıs University (TR)
- Ege University (TR)
- Directorate of Health (IS)
- Necmettin Erbakan University (TR)
- Istanbul University-Cerrahpaşa (TR)
- Sağlık Bilimleri Üniversitesi (TR)
- Lokman Hekim Üniversitesi (TR)
- Eskişehir Osmangazi University (TR)
- Mersin Üniversitesi (TR)
- Kocaeli Üniversitesi (TR)
- Cukurova University (TR)
- Gaziantep University (TR)
- Atatürk University (TR)
- Istanbul University (TR)
- Marmara University (TR)
- Erciyes University (TR)
- Pharmaceuticals and Medical Devices Agency (JP)
- Gazi University (TR)
Publication Details
- Journal
- Frontiers in Neurology
- Published
- 2025-03-27
- DOI
- https://doi.org/10.3389/fneur.2025.1541507
- Citations
- 3
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 4.88