Cargando…

Motor unit changes in children with symptomatic spinal muscular atrophy treated with nusinersen

OBJECTIVES: To elucidate the motor unit response to intrathecal nusinersen in children with symptomatic spinal muscular atrophy (SMA) using a novel motor unit number estimation technique. METHODS: MScanFit MUNE studies were sequentially undertaken from the abductor pollicis brevis muscle after stimu...

Descripción completa

Detalles Bibliográficos
Autores principales:	Kariyawasam, Didu, D'Silva, Arlene, Howells, James, Herbert, Karen, Geelan-Small, Peter, Lin, Cindy Shin-Yi, Farrar, Michelle Anne
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BMJ Publishing Group 2021
Materias:	Neuromuscular
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7803907/ https://www.ncbi.nlm.nih.gov/pubmed/33106369 http://dx.doi.org/10.1136/jnnp-2020-324254

Ejemplares similares

Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy
por: Strauss, Kevin A., et al.
Publicado: (2018)

Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy
por: Kariyawasam, Didu S. T., et al.
Publicado: (2019)

Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme
por: D'Silva, Arlene M, et al.
Publicado: (2021)

Identification of Novel CSF-Derived miRNAs in Treated Paediatric Onset Spinal Muscular Atrophy: An Exploratory Study
por: D’Silva, Arlene M., et al.
Publicado: (2023)

Non-neural phenotype of spinal and bulbar muscular atrophy: results from a large cohort of Italian patients
por: Querin, Giorgia, et al.
Publicado: (2016)

“We needed this”: perspectives of parents and healthcare professionals involved in a pilot newborn screening program for spinal muscular atrophy
por: Kariyawasam, Didu S.T., et al.
Publicado: (2021)

Nusinersen for spinal muscular atrophy
por: Wurster, Claudia D., et al.
Publicado: (2018)

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
por: D'Silva, Arlene M., et al.
Publicado: (2022)

Differentiating lower motor neuron syndromes
por: Garg, Nidhi, et al.
Publicado: (2017)

The influence of tone on proximal femoral and acetabular geometry in neuromuscular hip displacement: A comparison of cerebral palsy and spinal muscular atrophy
por: Ulusaloglu, Armagan Can, et al.
Publicado: (2022)

Nusinersen Treatment in Adults With Spinal Muscular Atrophy
por: Duong, Tina, et al.
Publicado: (2021)

An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials
por: Darras, Basil T., et al.
Publicado: (2019)

The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
por: Ricotti, Valeria, et al.
Publicado: (2016)

Clinical features and a mutation with late onset of limb girdle muscular dystrophy 2B
por: Takahashi, Toshiaki, et al.
Publicado: (2013)

Gene therapy-based strategies for spinal muscular atrophy—an Asia-Pacific perspective
por: Farrar, Michelle A., et al.
Publicado: (2023)

Incidence of Duchenne muscular dystrophy in the modern era; an Australian study
por: Kariyawasam, Didu, et al.
Publicado: (2022)

Validation of genetic modifiers for Duchenne muscular dystrophy: a multicentre study assessing SPP1 and LTBP4 variants
por: van den Bergen, Janneke C, et al.
Publicado: (2015)

Nusinersen as a Therapeutic Agent for Spinal Muscular Atrophy
por: Li, Qing
Publicado: (2020)

Nusinersen and Spinal Muscular Atrophies: Where are we in 2020?
por: Khadilkar, Satish V., et al.
Publicado: (2020)

Symptomatic intracranial hypertension in an adult patient with spinal muscular atrophy and arachnoid cysts receiving nusinersen
por: Machetanz, Gerrit, et al.
Publicado: (2023)

Imaging muscle as a potential biomarker of denervation in motor neuron disease
por: Jenkins, Thomas M, et al.
Publicado: (2018)

Fatigue in adults with spinal muscular atrophy under treatment with nusinersen
por: Kizina, K., et al.
Publicado: (2020)

Parents’ perspectives on nusinersen treatment for children with spinal muscular atrophy
por: van Kruijsbergen, Mette, et al.
Publicado: (2021)

Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study
por: Acsadi, Gyula, et al.
Publicado: (2021)

Nusinersen: A Review in 5q Spinal Muscular Atrophy
por: Hoy, Sheridan M.
Publicado: (2021)

Scoliosis in Spinal Muscular Atrophy Type 1 in the Nusinersen Era
por: Al Amrani, Fatima, et al.
Publicado: (2022)

Nusinersen mitigates neuroinflammation in severe spinal muscular atrophy patients
por: Nuzzo, Tommaso, et al.
Publicado: (2023)

Charcot-Marie-Tooth disease type 2CC due to NEFH variants causes a progressive, non-length-dependent, motor-predominant phenotype
por: Pipis, Menelaos, et al.
Publicado: (2022)

Nusinersen in type 0 spinal muscular atrophy: should we treat?
por: Tiberi, Eloisa, et al.
Publicado: (2020)

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy
por: Pera, Maria Carmela, et al.
Publicado: (2021)

Correction to: Nusinersen: A Review in 5q Spinal Muscular Atrophy
por: Hoy, Sheridan M.
Publicado: (2021)

History of development of the life-saving drug “Nusinersen” in spinal muscular atrophy
por: Qiu, Jiaying, et al.
Publicado: (2022)

Treatment with Ataluren for Duchene Muscular Dystrophy
por: Morkous, Sameh S.
Publicado: (2020)

Intrathecal nusinersen administration in adult spinal muscular atrophy patients with complex spinal anatomy
por: Cordts, Isabell, et al.
Publicado: (2020)

Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience
por: Haché, Manon, et al.
Publicado: (2016)

Feasibility and safety of intrathecal treatment with nusinersen in adult patients with spinal muscular atrophy
por: Stolte, Benjamin, et al.
Publicado: (2018)

Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US
por: Thokala, Praveen, et al.
Publicado: (2020)

Nusinersen Wearing-Off in Adult 5q-Spinal Muscular Atrophy Patients
por: Osmanovic, Alma, et al.
Publicado: (2021)

Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy
por: Osmanovic, Alma, et al.
Publicado: (2021)

Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy
por: Freigang, Maren, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_6u4ntu15hh0dmo8tlgc80urg1m