Results 41 to 50 of about 4,206 (188)

Comparison of Nusinersen and Evrysdi in the Treatment of Spinal Muscular Atrophy [PDF]

E3S Web of Conferences, 2021
Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disease that commonly affects children, and usually worsens with age that often leads to permanent disability and death for many of the SMA patients.
Zhu Xiaoying
doaj   +1 more source

Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia

Patient Preference and Adherence, 2021
Yazed AlRuthia,1,2 Ghadah S Almuaythir,1 Hala H Alrasheed,1 Wejdan R Alsharif,1 Mohamad-Hani Temsah,3,4 Fahad Alsohime,3,4 Ibrahim Sales,1 Monira Alwhaibi,1 Fahad A Bashiri3,5 1Department of Clinical Pharmacy, College of Pharmacy, King Saud University ...
AlRuthia Y, Almuaythir GS, H Alrasheed H, Alsharif WR, Temsah MH, Alsohime F, Sales I, Alwhaibi M, Bashiri FA   +8 more
doaj  

Assessment of motor function and nutritional status in children with spinal muscular atrophy treated with nusinersen after loading period in Western China: a retrospective study

BMC Neurology, 2023
Background Spinal muscular atrophy (SMA) is a progressive degenerative neuromuscular disease. Nusinersen, with its quick onset of action, can benefit patients early in the treatment course.
Hua Yang, Qiuji Tao, Dan Li, Jie Yang, Qianyun Cai, Jing Gan, Shaoping Huang, Rong Luo   +7 more
doaj   +1 more source

Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients

Brain Sciences, 2021
Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases.
Lars Hendrik Müschen, Alma Osmanovic, Camilla Binz, Konstantin F. Jendretzky, Gresa Ranxha, Paul Bronzlik, Omar Abu-Fares, Flavia Wiehler, Nora Möhn, Martin W. Hümmert, Stefan Gingele, Friedrich Götz, Martin Stangel, Thomas Skripuletz, Olivia Schreiber-Katz, Susanne Petri   +15 more
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Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model [PDF]

, 2022
Background: Nusinersen is approved for the treatment of spinal muscular atrophy. The most common approved dosing regimen is four intrathecal loading doses of nusinersen 12 mg; the first three are administered at 14-day intervals followed by a fourth dose
Berger, Zdenek, MacCannell, Drew, Iannaccone, Susan T, Finkel, Richard S, Muntoni, Francesco, Kirschner, Janbernd, Kuntz, Nancy L, Farrar, Michelle A, Mercuri, Eugenio, Valente, Marta   +9 more
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Nusinersen: A Review in 5q Spinal Muscular Atrophy

, 2021
DeclarationsFunding The preparation of this review was not supported by any external funding.Authorship and Conflict of interest Sheridan M. Hoy is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of ...
Sheridan M. Hoy (4897468)
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Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

, 2022
Limited evidence exists on real-world adherence to nusinersen for the treatment of spinal muscular atrophy (SMA). Data are presented from a multi-site retrospective chart review of 86 adults with SMA initiating nusinersen at nine US centers between ...
Michelson, David, Cartwright, Michael S., Proud, Crystal M., Martinez, Marta, Choi, Joseph M., McCormick, M. Eileen, Chandak, Aastha, Elman, Lauren, Khachatryan, Artak, Ajroud-Driss, Senda, Paradis, Angela D., Youn, Bora, Heiman-Patterson, Terry, Frey, Margaret R.   +13 more
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Clinical experience of nusinersen in a broad spectrum of spinal muscular atrophy: A retrospective study

Annals of Indian Academy of Neurology, 2020
Background: Nusinersen has recently been approved and more widely used as first-line treatment of spinal muscular atrophy (SMA). This study aimed to evaluate the real-world experience of nusinersen use for patients with a broad spectrum of SMA.
Ae Ryoung Kim, Jong-Mok Lee, Yu-Sun Min, Hoseok Lee, Dongsub Kim, Su-Kyeong Hwang, Soonhak Kwon, Yun Jeong Lee   +7 more
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Assessment of Bulbar Function in Adult Patients with 5q-SMA Type 2 and 3 under Treatment with Nusinersen

Brain Sciences, 2021
The antisense oligonucleotide nusinersen has been shown to improve trunk and limb motor function in patients with spinal muscular atrophy (SMA). Bulbar dysfunction, which is regularly present in SMA, is not captured by standard motor scores, and ...
Svenja Brakemeier, Benjamin Stolte, Andreas Thimm, Kathrin Kizina, Andreas Totzeck, Juan Munoz-Rosales, Christoph Kleinschnitz, Tim Hagenacker   +7 more
doaj   +1 more source

Assessment of respiratory muscles and motor function in children with SMA treated by nusinersen

, 2021
Introduction: Nusinersen is associated with an improvement in motor function in children with spinal muscular atrophy (SMA) but data on respiratory muscles strength are scarce.
Barnerias C, Durigneux J, Fauroux B, Amaddeo A, Bénézit A, Dabaj I, Carlier RY, Desguerre I, de la Banda MG, Khirani S, Pruvost S, Quijano-Roy S   +11 more
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