Results 151 to 160 of about 6,859 (228)

Development of gene therapies—lessons from nusinersen [PDF]

, 2017
Li Xu, Ilan Irony, Wilson W. Bryan, Billy Dunn   +3 more
openalex   +1 more source

Compound Muscle Action Potential (CMAP) Amplitude Trajectories and Pattern in Adults with 5q-Spinal Muscular Atrophy Receiving Nusinersen Therapy: A Multicenter, Binational Observational Study. [PDF]

Eur J Neurol
Bjelica B, Wohnrade C, Osmanovic A, Schreiber-Katz O, Koerner S, Kollewe K, Haeckl S, Freigang M, Cordts I, Becker B, Vogt C, Muhandes MT, Günther R, Deschauer M, Koch JC, Tuerk M, Regensburger M, Neuwirth C, Petri S.   +18 more
europepmc   +1 more source

Comparative efficacy of risdiplam and nusinersen in Type 2 and 3 spinal muscular atrophy patients: A cohort study using real-world data

Mahmoud Reza Ashrafi, Marzieh Babaee, Seyed Saeed Hashemi Nazari, Mohammad Barzegar, Mohammadreza Ghazavi, Mehran Beiraghi Toosi, Shahriar Nafissi, Soroor Inaloo, Gholamreza Zamani Ghaletaki, Farzad Fatehi, Ramin Heshmat, Masood Ghahvechi Akbari, Alireza Abdi, Hassan Bakhtiary, Hadi Montazerlotfelahi, Ali Abbaskhanian, Seyed Ahmad Hosseini, Hossein Farshadmoghadam, Seyyed Mohammad Mahdi Hosseiny, Fakhreddin Shariatmadari, Bentolhoda Ziaadini, Meisam Babaei, Azita Tavasoli, Sedighe Nikbakht, Ali Akbar Momen, Ali Khajeh, Vahid Aminzadeh, Mohsen Mollamohammadi, Mohammad Mehdi Taghdiri, Mohammad Mehdi Nasehi, Sara Memarian, Reza Shervin Badv, Morteza Heidari, Narjes Jafari   +33 more
openalex   +1 more source

Real-world evidence of Nusinersen treatment for patients with spinal muscular atrophy in the Kingdom of Saudi Arabia: Initial insights from the Saudi national spinal muscular atrophy program. [PDF]

Saudi Med J
Al-Jedai AH, Almudaiheem HY, Alaama TY, Alsaman ASS, Abdo Bashiri F, Almuhaizae MA, Alghamdi FA, Alsakran AA, Matter EAY, Ibrahim FZ.   +9 more
europepmc   +1 more source

A sensitive analytical strategy of oligonucleotide functionalized fluorescent probes for detection of nusinersen sodium in human serum

Yujuan Zhan, Jingru Guo, Penghui Hu, Ruiyan Huang, Jiangyue Ning, Xingyan Bao, Haotian Chen, Zelong Yan, Li Ding, Chang Shu   +9 more
openalex   +1 more source

A pediatric quantitative systems pharmacology model of neurofilament trafficking in spinal muscular atrophy treated with the antisense oligonucleotide nusinersen

, 2022
Alessio Paris, Pranami Bora, Silvia Parolo, Drew MacCannell, Michael Monine, Nick van der Munnik, Xiao Tong, Satish A. Eraly, Zdenek Berger, Danielle Graham, Toby A. Ferguson, Enrico Domenici, Ivan Nestorov, Luca Marchetti   +13 more
openalex   +1 more source

Five-year disease-modifying therapeutic experience of 102 Chinese paediatric 5q-spinal muscular atrophy: a retrospective analysis. [PDF]

Brain Commun
Jiang M, Liang C, Zhang Y, Zheng K, Xu K, He L, Tao J, Wu W, Zheng R, Rao M, Zhang W, Zhou W, Liu L.   +12 more
europepmc   +1 more source

Intrathecal nusinersen treatment in a Chinese patient with spinal muscular atrophy type 3 after ventriculo-peritoneal shunt placement: A case report and review of literature

Kai Ma, Yi Lu, Dong Wang, Xiao Han, Lei Liang, Yuanyuan Zhang   +5 more
openalex   +1 more source

A therapeutic antisense oligonucleotide encompassing 2'-<i>O</i>-methoxyethyl modification triggers unique perturbation of the transcriptome. [PDF]

NAR Mol Med
Ottesen EW, Murzyn WA, Kaas RL, Bertrand KJ, Payne JL, Singh RN.   +5 more
europepmc   +1 more source

Open-label phase IV trial evaluating nusinersen after onasemnogene abeparvovec in children with spinal muscular atrophy. [PDF]

J Clin Invest
Proud CM, Finkel RS, Parsons JA, Masson R, Brandsema JF, Kuntz NL, Foster R, Li W, Littauer R, Sohn J, Fradette S, Youn B, Paradis AD, RESPOND Study Group.   +13 more
europepmc   +1 more source