Results 111 to 120 of about 6,859 (228)

Nusinersen Treatment in Adults With Spinal Muscular Atrophy [PDF]

, 2021
Tina Duong, Connie Wolford, Michael McDermott, Chelsea E. Macpherson, Amy Pasternak, Allan M. Glanzman, William B. Martens, Elizabeth Kichula, Basil T. Darras, Darryl C. De Vivo, Zarazuela Zolkipli‐Cunningham, Richard S. Finkel, Michael Zeineh, Max Wintermark, Jacinda B. Sampson, Katharine A. Hagerman, Sally Dunaway Young, John Day   +17 more
openalex   +1 more source

Corrigendum to ‘Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen’

, 2022
Drew MacCannell, Zdenek Berger, Lilly East, Eugenio Mercuri, Janbernd Kirschner, Francesco Muntoni, Michelle A. Farrar, Joanna Peng, Jie Zhou, Ivan Nestorov, Wildon Farwell, Richard S. Finkel   +11 more
openalex   +1 more source

Safety analysis of laboratory parameters in paediatric patients with spinal muscular atrophy treated with nusinersen

BMC Pediatrics
Background Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that can be treated with intrathecal nusinersen, an antisense oligonucleotide.
Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li   +6 more
doaj   +1 more source

Clinical Implication of Dosimetry of Computed Tomography- and Fluoroscopy-Guided Intrathecal Therapy With Nusinersen in Adult Patients With Spinal Muscular Atrophy [PDF]

, 2019
Kathrin Kizina, Benjamin Stolte, Andreas Totzeck, Saskia Bolz, Michael Fleischer, Christoph Mönninghoff, Nika Guberina, Denise Oldenburg, Michael Forsting, Christoph Kleinschnitz, Tim Hagenacker   +10 more
openalex   +1 more source

Changes in electrophysiological findings of spinal muscular atrophy type I after the administration of nusinersen and onasemnogene abeparvovec: two case reports [PDF]

, 2023
Tomoko Mizuno, Tadashi Kanouchi, Yumie Tamura, Ko Hirata, Runa Emoto, Tomonori Suzuki, Kenichi Kashimada, Tomohiro Morio   +7 more
openalex   +1 more source

Cost-Effectiveness Analysis of Newborn Screening for Spinal Muscular Atrophy in Italy. [PDF]

Background and Objective Untreated spinal muscular atrophy (SMA) is the leading genetic cause of death in children younger than 2 years of age. Early detection through newborn screening allows for presymptomatic diagnosis and treatment of SMA.
Bischof M, Ghetti G, Marcellusi A, Mennini FS, Pane M., Pistillo GM   +5 more
core   +1 more source

Surgical correction of a ventricular septal defect in a child with spinal muscular atrophy type 2 treated with nusinersen sodium: a case report [PDF]

, 2023
Mehmet Biçer, Şima Kozan, Figen Öztürk, Ayfer Arduç Akçay   +3 more
openalex   +1 more source

Short review on determination of nusinersen for spinal muscular atrophy treatment [PDF]

Preprint artykułuNusinersen, an antisense oligonucleotide, is an active ingredient of the first drug approved by the Food and Drug Administration for the treatment of spinal muscular atrophy, a genetic condition characterized by progressive muscle ...
Balińska, Natalia, Ostrowska, Karolina, Studzińska, Sylwia   +2 more
core   +1 more source

The Impact of Nusinersen Treatment on Respiratory Function in Patients with Spinal Muscular Atrophy: A Systematic Review [PDF]

Mona Aldukain, Ali Aldukain, Assal Hobani, Amal Barakat, Lujain Alsayyed, Maher T Al-Omar, Maha Saad Zain Al-Abeden, Nora Alzoum, Ali Alsuheel Asseri   +8 more
openalex   +1 more source

Ultrasound assisted versus landmark based intrathecal administration of nusinersen in adults with spinal muscular atrophy disease: A randomized trial [PDF]

Bruno Antonio Zanfini, Stefano Catarci, Agata Katia Patanella, Francesco Vassalli, Luciano Frassanito, Marika Pane, Matteo Biancone, Mariangela Di Muro, Eleonora Rizzi, Eugenio Mercuri, Mario Sabatelli, Gaetano Draisci   +11 more
openalex   +1 more source