Results 191 to 200 of about 6,101 (249)

Switching from Nusinersen to Risdiplam in Spinal Muscular Atrophy: A Comparative Analysis of Safety, Efficacy, and Economic Impact. [PDF]

Hosp Pharm
Castellana E.
europepmc   +1 more source

MicroRNAs as Biomarkers in Spinal Muscular Atrophy. [PDF]

Biomedicines
Barbo M, Glavač D, Jezernik G, Ravnik-Glavač M.   +3 more
europepmc   +1 more source

Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis. [PDF]

Orphanet J Rare Dis
Patel A, Toro W, Yang M, Song W, Desai R, Ye M, Tabatabaeepour N, Dabbous O.   +7 more
europepmc   +1 more source

Correction: AlTawari et al. Nusinersen Treatment for Spinal Muscular Atrophy: Retrospective Multicenter Study of Pediatric and Adult Patients in Kuwait. Neurol. Int. 2024, 16, 631-642. [PDF]

Neurol Int
AlTawari A, Zakaria M, Kamel WA, Shaalan N, Elghazawi GAI, Ali MEA, Salota D, Attia A, Elanany EEA, Shalaby O, Alqallaf F, Mitic V, Bastaki L.   +12 more
europepmc   +1 more source

The expanding application of antisense oligonucleotides to neurodegenerative diseases. [PDF]

J Clin Invest
Sumner CJ, Miller TM.
europepmc   +1 more source

Nusinersen effectiveness and safety in pediatric patients with 5q-spinal muscular atrophy: a multi-center disease registry in China

Xiaoli Yao, Jing Peng, Rong Luo, Xiuxia Wang, Xinguo Lu, Liwen Wu, Ruifeng Jin, Jianmin Zhong, Jianmin Liang, Siqi Hong, Lin Yang, Xiaoli Zhang, Shanshan Mao, Jun Hu, Zhe Tao, Dan Sun, Hua Wang, Li Zhang, Yanyan Xia, Ken Chen, Yi Wang   +20 more
openalex   +1 more source

Proteomics profiling and machine learning in nusinersen-treated patients with spinal muscular atrophy. [PDF]

Cell Mol Life Sci
Panicucci C, Sahin E, Bartolucci M, Casalini S, Brolatti N, Pedemonte M, Baratto S, Pintus S, Principi E, D'Amico A, Pane M, Sframeli M, Messina S, Albamonte E, Sansone VA, Mercuri E, Bertini E, Sezerman U, Petretto A, Bruno C.   +19 more
europepmc   +1 more source

Experiences of Family Members and Patients with Spinal Muscular Atrophy Under the Multi-Level Medical Security System in Shaanxi Province, China: A Mixed Study. [PDF]

Healthcare (Basel)
Zhao Y, Ding S, Lin C, Han Y, Zhao M.
europepmc   +1 more source

The Relevance of Spinal Muscular Atrophy Biomarkers in the Treatment Era. [PDF]

Biomedicines
Maretina M, Koroleva V, Shchugareva L, Glotov A, Kiselev A.   +4 more
europepmc   +1 more source

A cross-sectional and longitudinal evaluation of serum creatinine as a biomarker in spinal muscular atrophy. [PDF]

Orphanet J Rare Dis
Zhao X, Gong Z, Luo H, Li Z, Gao R, Yang K, Deng W, Peng S, Ba L, Liu Y, Zhang M.   +10 more
europepmc   +1 more source