Results 21 to 30 of about 6,101 (249)

Nusinersen potentially effective in SMA [PDF]

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

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

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   +2 more sources

Profiling neuroinflammatory markers and response to nusinersen in paediatric spinal muscular atrophy [PDF]

Scientific Reports
Neuroinflammation is an emerging clinical feature in spinal muscular atrophy (SMA). Characterizing neuroinflammatory cytokines in cerebrospinal fluid (CSF) in SMA and their response to nusinersen is important for identifying new biomarkers and ...
Qiang Zhang, Ying Hong, Chiara Brusa, Mariacristina Scoto, Nikki Cornell, Parth Patel, Giovanni Baranello, Francesco Muntoni, Haiyan Zhou   +8 more
doaj   +2 more sources

Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients

European Journal of Neurology, 2022
Yoram Nevo, Iddo Magen, Sharon Aharoni
exaly   +3 more sources

Intrathecal administration of nusinersen in adult and adolescent patients with spinal muscular atrophy and scoliosis: Transforaminal versus conventional approach [PDF]

, 2019
Sara Bortolani, G. Stura, Giancarlo Ventilii, Liliana Vercelli, Enrica Rolle, Federica Ricci, Mauro Bergui, Tiziana Mongini   +7 more
openalex   +3 more sources

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy [PDF]

New England Journal of Medicine, 2018
Eugenio Mercuri, Basil T Darras, Claudia A Chiriboga   +2 more
exaly   +2 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

History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy

Frontiers in Cellular Neuroscience, 2022
Spinal muscular atrophy (SMA) is an autosomal recessive disorder with an incidence of 1/6,000–1/10,000 and is the leading fatal disease among infants. Previously, there was no effective treatment for SMA.
Jiaying Qiu, Jiaying Qiu, Liucheng Wu, Ruobing Qu, Tao Jiang, Jialin Bai, Lei Sheng, Pengchao Feng, Junjie Sun   +8 more
doaj   +1 more source

Transitioning From Nusinersen to Risdiplam for Spinal Muscular Atrophy in Clinical Practice: A Single-Center Experience. [PDF]

Muscle Nerve
ABSTRACT Background Nusinersen and risdiplam are U.S. Food and Drug Administration (FDA)‐approved treatments for spinal muscular atrophy (SMA). No head‐to‐head clinical trials to assess efficacy exist. Observational studies are needed to determine if transitioning to risdiplam is safe and efficacious.
Bekircan-Kurt CE, Subramanian S, Chagat S, Mackenzie SJ, Iammarino M, Reash N, Richardson C, Tsao CY, Noritz G, Gushue C, Kotha K, Paul G, Shell R, Alfano LN, Lowes LP, Connolly AM, Waldrop MA.   +16 more
europepmc   +2 more sources

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, Farrar, Michelle A, Finkel, Richard S, Iannaccone, Susan T, Kirschner, Janbernd, Kuntz, Nancy L, MacCannell, Drew, Mercuri, Eugenio, Muntoni, Francesco, Valente, Marta   +9 more
core   +1 more source