Results 181 to 190 of about 7,657 (243)

Spinal presentations in children with type 1 spinal muscular atrophy on nusinersen treatment across the SMA-REACH UK network: a retrospective national observational study

BMJ Open
Background Prior to the introduction of disease-modifying treatments (DMTs), children with type 1 spinal muscular atrophy (SMA) typically did not survive beyond the age of 2 years; management was mainly palliative.
L. Abbott, Marion Main, Amy Wolfe, A. Rohwer, G. Baranello, P. Munot, Adnan Y. Manzur, F. Muntoni, M. Scoto   +8 more
semanticscholar   +1 more source

Nusinersen

Reactions Weekly, 2021
  +6 more sources

Nusinersen (Spinraza)

Canadian Journal of Health Technologies, 2022
CADTH recommends that Spinraza should not be reimbursed by public drug plans for the treatment of patients with type II and type III 5q spinal muscular atrophy (SMA) regardless of ambulatory status if initiated in patients older than 18 years of age. No randomized clinical trials evaluating the efficacy or safety of Spinraza in treatment ...
openaire   +1 more source

Motor function and compound muscle action potential amplitude in children with spinal muscular atrophy treated with nusinersen.

Brain & development (Tokyo. 1979)
BACKGROUND Disease-modifying therapies can improve motor function in patients with spinal muscular atrophy (SMA), but efficacy varies between individuals. The aim was to evaluate the efficacy and safety of nusinersen treatment in children with SMA and to
Yin Peng, Lianying Feng, Jinfeng Wu, Qianyun Zhou, Hailang Liu, Jin Chen, Xiao-jie Song, Wei Han, Fuyi Zhang, Ping Yuan, Zheng-xiong Yao, Lingling Xie, Mei Li, Li Jiang, Siqi Hong   +14 more
semanticscholar   +1 more source

Mechanisms of functional improvement behind nusinersen treatment in adult spinal muscular atrophy.

Experimental Neurology
Nusinersen treatment not only prevents neurological deterioration in presymptomatic or early symptomatic children with spinal muscular atrophy (SMA) but promotes functional improvement in the later plateau phase in adults with SMA, though the mechanisms ...
P. Hsieh, Hsing-Jung Lai, Yih-Chih Kuo, Chih-Chao Yang, Po-Ya Huang, Chen-Hung Ting, Shao-Ting Tai, Chia-Hsin Kao, Yi-Chieh Tsai, Hsi-Wen Huang, J. Shieh, Han Chiou, Lo-Fan Jeng, Wen-Chin Weng, Li-Kai Tsai   +14 more
semanticscholar   +1 more source

Systematic Review and Meta-analysis of Long-Term Nusinersen Effectiveness in Adolescents and Adults with Spinal Muscular Atrophy

Advances in Therapy
Given the lifelong progression of spinal muscular atrophy (SMA), understanding the long-term effects of nusinersen treatment is crucial. Prior systematic literature reviews (SLRs) consolidated evidence on the real-world effectiveness of nusinersen in ...
T. Hagenacker, Angela D. Paradis, Katherine A Lawson-Michod, Bora Youn   +3 more
semanticscholar   +1 more source

Longitudinal Assessment of 4‐Year HFMSE Changes in SMA II and III Patients Treated With Nusinersen

European Journal of Neurology
The aim of this international retrospective study was to assess 4‐year change using the Hammersmith Functional Motor Scale Expanded (HFMSE) in individuals with type II and III spinal muscular atrophy (SMA) treated with nusinersen and to establish ...
Giorgia Coratti, F. Bovis, M. Pane, A. Pasternak, Emilio Albamonte, I. Mizzoni, A. Glanzman, S. Morando, Jacqueline Montes, I. Cavallina, S. Dunaway Young, Tina Duong, Enrica Rolle, M. Civitello, R. de Sanctis, C. Bravetti, Federica Ricci, Giulio Gadaleta, T. Mongini, M. Sframeli, M. Pera, S. Messina, Adele D’Amico, M. Catteruccia, Noemi Brolatti, Michio Hirano, Z. Zolkipli-Cunningham, Basil T. Darras, E. Bertini, Claudio Bruno, John W. Day, V. Sansone, Richard S. Finkel, Eugenio Mercuri   +33 more
semanticscholar   +1 more source

Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy.

Clinical neurology and neurosurgery (Dutch-Flemish ed. Print)
INTRODUCTION Current disease-modifying treatments for spinal muscular atrophy (SMA) have been shown to significantly improve the course of the disease, but data on long-term real-world outcomes remain scarce.
A. Tscherter, Leonie Steiner, P. Broser, C. Enzmann, Elea Galiart, Bettina C. Henzi, D. Jacquier, A. Mathis, C. Neuwirth, G. P. Ramelli, P. Ripellino, Olivier Scheidegger, B. Schreiner, E. I. Schwarz, G. M. Stettner, A. Klein   +15 more
semanticscholar   +1 more source

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

Frontiers in Neurology
Background SMA 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.
Ömer Bektaş, Murat Gülşen, O. Dursun, Ahmet Tekin, Deniz Yüksel, Ercan Demir, Gülten Öztürk, Sema Saltık, Özlem Hergüler, Ayşe Aysima Özçelik, Hüseyin Tan, B. Özgör, A. Ekici, M. Yüksel, Suleyman Sahın, Ö. Duman, M. Kömür, F. Baydan, Edibe Pembegül Yıldız, Bülent Kara, U. Yiş, Seda Kanmaz, K. Carman, E. Arslan, M. Canpolat, A. Güven, Gökçen Öztuncer, A. Ünalp, D. Ardıçlı, A. Karaduman, G. Zararsiz, G. Deda, Ömer Murat Onur Ahmet Deniz Ercan Gülten Sema Özlem Ayş Bektaş Gülşen Burak Dursun Tekin Yüksel Demir Öztü, Ömer Bektaş, Murat Gülşen, Onur Burak Dursun, Ahmet Tekin, Deniz Yüksel, Ercan Demir, Gülten Öztürk, Sema Saltık, Özlem Hergüler, Ayşe Aysima Özçelik, Hüseyin Tan, B. Özgör, A. Ekici, Merve Feyza Yüksel, Suleyman Sahın, Ö. Duman, M. Kömür, F. Baydan, E. Pembegül Yıldız, Bülent Kara, U. Yiş, Seda Kanmaz, Kursat Bora Carman, E. Acar Arslan, M. Canpolat, Ahmet Sami Güven, Gökçen Öztuncer, A. Ünalp, D. Ardıçlı, Aynur Ayşe Karaduman, G. Zararsiz, Nurşah Yeniay Süt, O. Ünver, Tuğçe Damla Dilek, Eif Yıldırım, F. Incecik, Meral Karadağ, Mehmet Fatih Bütün, Burçin Gönüllü Polat, Yiğithan Güzin, Mehmet Akif KILIC, Adnan Deniz, Gamze Sarıkaya Uzan, Sanem Yılmaz Keskin, Fatih M. Özdemir, Ç. Yarar, Nihal Yıldız, H. Acer, Hüseyin Çaksen, S. Aydin, P. Karaoğlu, Ayşe Neşe Çıtak, T. Kendirli, Birkan Sonel Tur, Gülcan Akyüz, Ceren Bibinoğlu Amirov, Gülen Gül Mert, Mahmut Aslan, Ş. Haspolat, Özlem Ersoy, Osman Kipoğlu, Defne Alikılıç, H. Tekgül, Ülkühan Kaya, Arife Derda Yücel Şen, Pınar Özkan Kart, H. Per, Ayşe Aksoy, Ünsal Yılmaz, S. Teber, Özben Akıncı Göktaş, D. Türkdoğan, Fitnat Uluğ, S. Bilge, Orhan Coşkun, Merve Öztürk, Esra Atmaca, Ali Cansu, Hakan Gümüş, E. Karadağ Saygı, Ö. Karaca, S. Güngör, N. Çobanoğlu, Melek Sezgin, M. Yıldırım, Yasemin Gökdemir, Esma Şengenç, Yılmaz Zindar, Özlem Yayıcı, Serkan Köken, M. Güngör, S. Kırık, Meltem Çobanoğulları Direk, G. Deda   +126 more
semanticscholar   +1 more source

LC-MS/MS quantification of nusinersen in rat cerebrospinal fluid and preclinical pharmacokinetics study application.

Bioanalysis
BACKGROUND Background: An oligonucleotide drug named nusinersen sodium is used to treat Spinal Muscular Atrophy (SMA), requires accurate detection for therapeutic research. There are no published reports on liquid chromatography-tandem mass spectrometry (
Yujie Li, Shu Zhang, Xiayi Wang, Xiaochuan Li, Lizhong Guo   +4 more
semanticscholar   +1 more source