Results 191 to 200 of about 7,657 (243)

Nusinersen

Reactions Weekly, 2022
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Difficulty of administration of nusinersen in complex-column spinal muscular atrophy: New alternative technique by means of cervical intrathecal access through an Ommaya reservoir.

European journal of paediatric neurology
BACKGROUND The study aimed to describe a new Ommaya reservoir implantation method in late-onset SMA patients, assessing its safety and effectiveness under standard clinical conditions. METHODS Prospective observational study.
B. Fernández, J. P. Paz Solís, M. T. G. Romero, Miguel Ángel Fernández García, María Román de Aragón, Fernando Carceller Benito, S. I. Pascual Pascual   +6 more
semanticscholar   +1 more source

Nusinersen

Reactions Weekly, 2023
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Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen

Developmental Medicine & Child Neurology
To describe bulbar function trajectories in patients with spinal muscular atrophy (SMA) type 1 treated with nusinersen in the UK and Italy.
Georgia Stimpson, L. Fanelli, Eleanor Conway, Emily Johnson, B. Berti, M. Scoto, F. Muntoni, E. Mercuri, G. Baranello   +8 more
semanticscholar   +1 more source

Cerebrospinal fluid metabolomics reveals predictive biomarkers of nusinersen therapy efficacy in type II and type III spinal muscular atrophy patients

Neurological Sciences
This study investigated the predictive value of clinical characteristics and cerebrospinal fluid (CSF) metabolites for nusinersen efficacy in children with spinal muscular atrophy (SMA). In this study, clinical data and CSF samples were collected.
Dan Li, Na Sun, Hongbo Wu, Xueying Wang, Yongjing Shi, Lin Yang, Shaoping Huang, Kun Zhang, Changhong Yang   +8 more
semanticscholar   +1 more source

Nusinersen: First Global Approval

Drugs, 2017
Spinal muscular atrophy (SMA) is a rare autosomal recessive disorder characterized by muscle atrophy and weakness resulting from motor neuron degeneration in the spinal cord and brainstem. It is most commonly caused by insufficient levels of survival motor neuron (SMN) protein (which is critical for motor neuron maintenance) secondary to deletions or ...
openaire   +2 more sources

Enhancing the effects of nusinersen with cybernic treatment using Hybrid Assistive Limb (HAL) in spinal muscular atrophy: a real-world case series and exploratory cohort analysis

Orphanet Journal of Rare Diseases
Nusinersen therapy for spinal muscular atrophy (SMA) provides significant functional improvement when initiated pre-symptomatically or early in life. However, challenges remain in diverse populations with longer disease duration.
Takashi Nakajima, Toshio Saito, A. Hashiguchi, Taiki Nakabayashi, K. Kodera, K. Utsumi, Takeshi Kanayama, Haruka Urabe, S. Kinoshita   +8 more
semanticscholar   +1 more source

Nusinersen: A Treatment for Spinal Muscular Atrophy

Annals of Pharmacotherapy, 2018
Objective: To review the efficacy and safety of nusinersen (Spinraza) in the treatment of spinal muscular atrophy (SMA). Data Sources: An English-language literature search of PubMed and MEDLINE (1946 to June 2018) was performed using the terms nusinersen, ISIS-SMN (Rx), and spinal muscular atrophy.
Melanie K, Claborn, Debra L, Stevens, Cheri K, Walker, Brooke L, Gildon   +3 more
openaire   +2 more sources

Experience of nusinersen treatment in advanced spinal muscular atrophy type 1: Characteristics of late responders with delayed treatment efficacy.

European journal of paediatric neurology
OBJECTIVE Little clinical data is available for advanced cases of spinal muscular atrophy (SMA) type 1, particularly those requiring ventilation support.
Sachi Tokunaga, H. Shimomura, Takuya Horibe, N. Taniguchi, Tomoko Lee, Yasuhiro Takeshima   +5 more
semanticscholar   +1 more source

Considerations for repetitive intrathecal procedures in long-term nusinersen treatment for non-ambulatory spinal muscular atrophy

Scientific Reports
Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder characterized by progressive motor function loss and skeletal muscular atrophy. Nusinersen, an antisense oligonucleotide, is the first FDA-approved therapy to achieve a significant ...
H. Lee, Hyunjoo Lee, Young-Mock Lee, Sungjun Kim   +3 more
semanticscholar   +1 more source