Results 11 to 20 of about 6,290 (252)

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   +3 more sources

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

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

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

Respiratory Follow-up of Patients Treated with Nusinersen

Turkish Thoracic Journal, 2019
Introduction and Aim: We aimed to evaluate respiratory support need in patients treated with Nusinersen and to report their respiratory follow-up during the treatment period. Material and Methods: The study included patients treated with 4 and more doses of Nusinersen.
Pınar Ergenekon, Cansu Yılmaz Yeğit, Ela Erdem Eralp, Yasemin Gökdemir, G. Thomas, Olcay Ünver, Ayca Evkaya, Evrim Karadağ‐Saygı, Dilşad Türkdoğan, Bülent Karadağ   +9 more
openalex   +3 more sources

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
doaj   +2 more sources

Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy [PDF]

Orphanet Journal of Rare Diseases, 2021
Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse.
Maren Freigang, Petra Steinacker, Claudia Diana Wurster, Olivia Schreiber-Katz, Alma Osmanovic, Susanne Petri, Jan Christoph Koch, Kevin Rostásy, Björn Falkenburger, Albert Christian Ludolph, Markus Otto, Andreas Hermann, René Günther   +12 more
doaj   +2 more sources

Full-Length SMN Transcript in Extracellular Vesicles as Biomarker in Individuals with Spinal Muscular Atrophy Type 2 Treated with Nusinersen [PDF]

, 2023
Three therapeutic strategies have radically changed the therapeutic scenario for spinal muscular atrophy (SMA). However, therapeutic response differs between individuals.
Alcolea, Daniel, Alias, Laura, Badosa, C., Balsells, S., Bernal, Sara, Carrera-García, L., Codina, A., Expósito-Escudero, J., Jimenez-Mallebrera, C., Medina, J., Nascimento, A., Natera-de Benito, Daniel, Ortez, C., Torres Alcalá, Soraya, Trifunov, S., Universitat Autònoma de Barcelona   +15 more
core   +3 more sources

Nusinersen potentially effective in SMA [PDF]

Nature Reviews Neurology, 2016
Louise Adams
openalex   +3 more sources

Evaluation of inpatient and emergency department healthcare resource utilization and costs pre- and post-nusinersen for the treatment of spinal muscular atrophy using United States claims

Journal of Comparative Effectiveness Research
Aim: Nusinersen, administered by intrathecal injection at a dose of 12 mg, is indicated across all ages for the treatment of spinal muscular atrophy (SMA).
Cong Zhu, Craig Zaidman, Bora Youn, Angela D Paradis, Stephanie Raynaud, Bridget A Neville, Nicole B Johnson   +6 more
doaj   +2 more sources