Results 11 to 20 of about 6,101 (249)

Scientific rationale for a higher dose of nusinersen

Annals of Clinical and Translational Neurology, 2022
Objective The long‐term favorable safety profile of nusinersen provides an opportunity to consider a higher dose. We report on the relationships between nusinersen cerebrospinal fluid (CSF) exposure, biomarker levels, and clinical efficacy.
Richard S. Finkel, Monique M. Ryan, Samuel Ignacio Pascual Pascual, John W. Day, Eugenio Mercuri, Darryl C. De Vivo, Richard Foster, Jacqueline Montes, Juliana Gurgel‐Giannetti, Drew MacCannell, Zdenek Berger   +10 more
doaj   +4 more sources

Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China [PDF]

BMC Pediatrics
Background Nusinersen was the first approved disease modifying therapy (DMT) for spinal muscular atrophy (SMA). Intrathecal administration of nusinersen enables drug delivery directly to the central nervous system, where the motor neurons are located ...
Jing Peng, Xiaoli Yao, Rong Luo, Xiuxia Wang, Liwen Wu, Jianmin Zhong, Ruifeng Jin, Xinguo Lu, Jianmin Liang, Siqi Hong, Lin Yang, Xiaoli Zhang, Shanshan Mao, Zhe Tao, Jun Hu, Dan Sun, Hua Wang, Li Zhang, Yanyan Xia, Ken Chen, Yi Wang   +20 more
doaj   +2 more sources

Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study [PDF]

Heliyon
Background: The drug nusinersen is applied to improve motor function in patients with spinal muscle atrophy (SMA). However, research on the effects of this treatment on lung function is lacking.
Jicai Zhu, Xiaofang Chen, Haoke Sang, Minming Ma, Chunhui Tang   +4 more
doaj   +2 more sources

Successful pregnancy of an SMA type 3 sitter on Nusinersen therapy - a case report [PDF]

BMC Neurology
Background Due to improved treatment options, more SMA patients reach childbearing age. Currently, limited data on pregnant SMA patients is available, especially in relation to disease-modifying therapies (DMT).
Miriam Hiebeler, Simone Thiele, Maggie C. Walter   +2 more
doaj   +2 more sources

Routine Cerebrospinal Fluid Cytology Reveals Unique Inclusions in Macrophages During Treatment With Nusinersen

Frontiers in Neurology, 2019
Background: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder characterized by degeneration of spinal motor neurons leading to muscular weakness.
Stefan Gingele, Martin W Hümmert, Philipp Schwenkenbecher   +2 more
exaly   +3 more sources

Efficacy of Nusinersen Treatment in Type 1, 2, and 3 Spinal Muscular Atrophy: Real-World Data from a Single-Center Study [PDF]

Neurology International
Background: Spinal muscular atrophy (SMA) is an inherited neuromuscular disease characterized by progressive muscle weakness and atrophy due to the absence of the survival motor neuron 1 (SMN1) gene.
Anna Lemska, Piotr Ruminski, Jakub Szymarek, Sylwia Studzinska, Maria Mazurkiewicz-Beldzinska   +4 more
doaj   +2 more sources

Development of gene therapies—lessons from nusinersen [PDF]

Gene Therapy, 2017
The nusinersen development and approval process provide important lessons regarding the pathway to marketing approval for gene therapies. These lessons emphasize rigorous clinical trial design, flexibility in trial design and analysis, a collaborative effort with regular communications between the drug developer and the Food and Drug Administration ...
Li Xu, Ilan Irony, Wilson W. Bryan, Billy Dunn   +3 more
openalex   +4 more sources

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials [PDF]

CNS Drugs, 2019
Basil T Darras, Eugenio Mercuri, Ishir Bhan   +2 more
exaly   +2 more sources

Nusinersen Treatment in Adults With Spinal Muscular Atrophy [PDF]

Neurology Clinical Practice, 2021
To determine changes in motor and respiratory function after treatment with nusinersen in adults with spinal muscular atrophy (SMA) during the first two years of commercial availability in the USA.Data were collected prospectively on adult (age >17 years at treatment initiation) SMA participants in the Pediatric Neuromuscular Clinical Research (PNCR ...
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   +4 more sources

Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy

Therapeutic Advances in Neurological Disorders, 2018
Background: Nusinersen is an intrathecally administered antisense oligonucleotide (ASO) and the first approved drug for the treatment of spinal muscular atrophy (SMA).
Benjamin Stolte, Saskia Bolz, Christoph Kleinschnitz   +2 more
exaly   +2 more sources