Cargando…

Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA

Objective: To determine the safety and tolerability of nusinersen treatment in ambulatory adults with spinal muscular atrophy (SMA) and investigate the treatment effect on muscle strength, physical function, and motor unit physiology. Methods: Individuals aged 18 years or older with genetically conf...

Descripción completa

Detalles Bibliográficos
Autores principales:	Elsheikh, Bakri, Severyn, Steven, Zhao, Songzhu, Kline, David, Linsenmayer, Matthew, Kelly, Kristina, Tellez, Marco, Bartlett, Amy, Heintzman, Sarah, Reynolds, Jerry, Sterling, Gary, Weaver, Tristan, Rajneesh, Kiran, Kolb, Stephen J., Arnold, W. David
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Neurology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8174580/ https://www.ncbi.nlm.nih.gov/pubmed/34093395 http://dx.doi.org/10.3389/fneur.2021.650535

Ejemplares similares

Safety, Tolerability, and Effect of Nusinersen in Non-ambulatory Adults With Spinal Muscular Atrophy
por: Elsheikh, Bakri, et al.
Publicado: (2021)

Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen
por: Arnold, W David, et al.
Publicado: (2021)

Quality of Life in SMA Patients Under Treatment With Nusinersen
por: Mix, Lucas, et al.
Publicado: (2021)

Evaluator Training and Reliability for SMA Global Nusinersen Trials(1)
por: Glanzman, Allan M., et al.
Publicado: (2018)

Response of plasma microRNAs to nusinersen treatment in patients with SMA
por: Zaharieva, Irina T., et al.
Publicado: (2022)

NFL is a marker of treatment response in children with SMA treated with nusinersen
por: Olsson, Bob, et al.
Publicado: (2019)

Neurochemical markers in CSF of adolescent and adult SMA patients undergoing nusinersen treatment
por: Wurster, Claudia D., et al.
Publicado: (2019)

Cognitive performance of adult patients with SMA before and after treatment initiation with nusinersen
por: Vidovic, Maximilian, et al.
Publicado: (2023)

Circulating MyomiRs as Potential Biomarkers to Monitor Response to Nusinersen in Pediatric SMA Patients
por: Bonanno, Silvia, et al.
Publicado: (2020)

Muscle quantitative MRI in adult SMA patients on nusinersen treatment: a longitudinal study
por: Gallone, Annamaria, et al.
Publicado: (2022)

Correction: Cognitive performance of adult patients with SMA before and after treatment initiation with nusinersen
por: Vidovic, Maximilian, et al.
Publicado: (2023)

Preliminary insights into RNA in CSF of pediatric SMA patients after 6 months of nusinersen
por: Garofalo, M., et al.
Publicado: (2023)

Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany
por: Pechmann, Astrid, et al.
Publicado: (2018)

Muscle MRI in two SMA patients on nusinersen treatment: A two years follow-up
por: Barp, Andrea, et al.
Publicado: (2020)

Routine Cerebrospinal Fluid (CSF) Parameters in Patients With Spinal Muscular Atrophy (SMA) Treated With Nusinersen
por: Wurster, Claudia D., et al.
Publicado: (2019)

Successful weaning from mechanical ventilation in a patient with SMA type 1 treated with nusinersen
por: Lee, Jiwon, et al.
Publicado: (2021)

Type I SMA “new natural history”: long‐term data in nusinersen‐treated patients
por: Pane, Marika, et al.
Publicado: (2021)

Effect of the COVID-19 Pandemic on Children With SMA Receiving Nusinersen: What Is Missed and What Is Gained?
por: Agosto, Caterina, et al.
Publicado: (2021)

Biochemical and clinical biomarkers in adult SMA 3–4 patients treated with nusinersen for 22 months
por: De Wel, Bram, et al.
Publicado: (2022)

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy
por: Scheijmans, Féline E. V., et al.
Publicado: (2022)

Healthcare utilisation in children with SMA type 1 treated with nusinersen: a single centre retrospective review
por: Ali, Imran, et al.
Publicado: (2019)

PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice
por: Kordala, Anna J, et al.
Publicado: (2023)

Assessment of Bulbar Function in Adult Patients with 5q-SMA Type 2 and 3 under Treatment with Nusinersen
por: Brakemeier, Svenja, et al.
Publicado: (2021)

Evaluation of putative CSF biomarkers in paediatric spinal muscular atrophy (SMA) patients before and during treatment with nusinersen
por: Johannsen, Jessika, et al.
Publicado: (2021)

Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis
por: Coratti, Giorgia, 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)

Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study
por: Walter, Maggie C., et al.
Publicado: (2019)

Successful weaning from the invasive respiratory support after nusinersen treatment in a child with SMA type 1: A case report
por: Pan, Meiling, et al.
Publicado: (2023)

Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
por: Audic, Frédérique, et al.
Publicado: (2020)

Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model
por: Berciano, María T., et al.
Publicado: (2020)

Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!
por: Bartlett, Amy, et al.
Publicado: (2018)

A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale
por: Staunton, Hannah, et al.
Publicado: (2023)

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

Improved upper limb function in non-ambulant children with SMA type 2 and 3 during nusinersen treatment: a prospective 3-years SMArtCARE registry study
por: Pechmann, Astrid, et al.
Publicado: (2022)

Quality of Life Outcomes According to Differential Nusinersen Exposure in Pediatric Spinal Muscular Atrophy
por: Weaver, Meaghann S., et al.
Publicado: (2021)

The effectiveness of styrene-maleic acid (SMA) copolymers for solubilisation of integral membrane proteins from SMA-accessible and SMA-resistant membranes
por: Swainsbury, David J.K., et al.
Publicado: (2017)

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

Scientific rationale for a higher dose of nusinersen
por: Finkel, Richard S., et al.
Publicado: (2022)

SMA CARNI-VAL TRIAL PART II: A Prospective, Single-Armed Trial of L-Carnitine and Valproic Acid in Ambulatory Children with Spinal Muscular Atrophy
por: Kissel, John T., et al.
Publicado: (2011)

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

Cannot write session to /tmp/vufind_sessions/sess_n4e1tqaf4ri8m1527eq9cmnh6f