Nusinersen for children with type I spinal muscular atrophy: 4 years’ clinical experience in Turkish cohort

Nusinersen for children with type I spinal muscular atrophy: 4 years’ clinical experience in Turkish cohort

BackgroundSMA Type 1 is the most severe form of spinal muscular atrophy with early symptom onset, limited motor development, and poor prognosis. Recent genetic-based therapies, such as nusinersen, have transformed disease outcomes. We aimed to evaluate the long-term effects of nusinersen on motor, b...

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Main Authors: Ömer Bektaş, Murat Gülşen, Onur Burak Dursun, Ahmet Tekin, Deniz Yüksel, Ercan Demir, Gülten Öztürk, Sema Saltık, Özlem Hergüler, Ayşe Aysima Özçelik, Hüseyin Tan, Bilge Özgör, Arzu Ekici, Merve Feyza Yüksel, Süleyman Şahin, Özgür Duman, Mustafa Kömür, Figen Baydan, Edibe Pembegül Yıldız, Bülent Kara, Uluç Yiş, Seda Kanmaz, Kürşat Bora Çarman, Elif Acar Arslan, Mehmet Canpolat, Ahmet Sami Güven, Gökçen Öztuncer, Aycan Ünalp, Didem Ardıçlı, Aynur Ayşe Karaduman, Gökmen Zararsız, Gülhis Deda, Turkish SMA Study Group, Nurşah Yeniay Süt, Olcay Ünver, Tuğçe Damla Dilek, Eif Yıldırım, Faruk İncecik, Meral Karadağ, Mehmet Fatih Bütün, Burçin Gönüllü Polat, Yiğithan Güzin, Mehmet Akif Kılıç, Adnan Deniz, Gamze Sarıkaya Uzan, Sanem Yılmaz Keskin, Fatih M. Özdemir, Coşkun Yarar, Nihal Yıldız, Hamit Acer, Hüseyin Çaksen, Seren Aydın, Pakize Karaoğlu, Ayşe Neşe Çıtak, Tanıl Kendirli, Birkan Sonel Tur, Gülcan Akyüz, Ceren Bibinoğlu Amirov, Gülen Gül Mert, Mahmut Aslan, Şenay Haspolat, Özlem Ersoy, Osman Kipoğlu, Defne Alikılıç, Hasan Tekgül, Ülkühan Kaya, Arife Derda Yücel Şen, Pınar Özkan Kart, Hüseyin Per, Ayşe Aksoy, Ünsal Yılmaz, Serap Teber, Özben Akıncı Göktaş, Dilşad Türkdoğan, Fitnat Uluğ, Serap Bilge, Orhan Coşkun, Merve Öztürk, Esra Atmaca, Ali Cansu, Hakan Gümüş, Evrim Karadağ Saygı, Ömer Karaca, Serdal Güngör, Nazan Çobanoğlu, Melek Sezgin, Miraç Yıldırım, Yasemin Gökdemir, Esma Şengenç, Yılmaz Zindar, Özlem Yayıcı, Serkan Köken, Mesut Güngör, Serkan Kırık, Meltem Çobanoğulları Direk
Format: Article
Language:English
Published: Frontiers Media S.A. 2025-03-01
Series:Frontiers in Neurology
Subjects:
spinal muscular atrophy
SMA type 1
motor function
severe symptomatic
nusinersen
ventilatory
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2025.1541507/full
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https://www.frontiersin.org/articles/10.3389/fneur.2025.1541507/full

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