Cargando…

Muscle quantitative MRI in adult SMA patients on nusinersen treatment: a longitudinal study

The recent approval of disease-modifying therapies for spinal muscular atrophy (SMA) raised the need of alternative outcome measures to evaluate treatment efficacy. In this study, we investigated the potential of muscle quantitative MRI (qMRI) as a biomarker of disease progression in adult SMA3 pati...

Descripción completa

Detalles Bibliográficos
Autores principales:	Gallone, Annamaria, Mazzi, Federica, Bonanno, Silvia, Zanin, Riccardo, Moscatelli, Marco, Aquino, Domenico, Maggi, Lorenzo
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Pacini Editore Srl 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9237750/ https://www.ncbi.nlm.nih.gov/pubmed/35832502 http://dx.doi.org/10.36185/2532-1900-074

Ejemplares similares

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

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

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

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)

Safety, Tolerability, and Effect of Nusinersen Treatment in Ambulatory Adults With 5q-SMA
por: Elsheikh, Bakri, et al.
Publicado: (2021)

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

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

Identification of a cytokine profile in serum and cerebrospinal fluid of pediatric and adult spinal muscular atrophy patients and its modulation upon nusinersen treatment
por: Bonanno, Silvia, et al.
Publicado: (2022)

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)

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)

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)

Interval between contrast administration and T1-weighted MRI for cerebral adrenoleukodystrophy: a single-case observation
por: Moscatelli, Marco, 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)

PLIN4-related myopathy: clinical, histological and imaging data in a large cohort of patients
por: Maggi, Lorenzo, et al.
Publicado: (2023)

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)

Dysregulation of Muscle-Specific MicroRNAs as Common Pathogenic Feature Associated with Muscle Atrophy in ALS, SMA and SBMA: Evidence from Animal Models and Human Patients
por: Malacarne, Claudia, et al.
Publicado: (2021)

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)

MRI correlates of motoneuron loss in SMA
por: Sprenger-Svačina, Alina, et al.
Publicado: (2022)

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

Affection of Respiratory Muscles in ALS and SMA
por: Hermann, Wiebke, et al.
Publicado: (2022)

Longitudinal Quantitative MRI Evaluation of Muscle Involvement in Amyotrophic Lateral Sclerosis
por: Paoletti, Matteo, et al.
Publicado: (2021)

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)

Reply to Chacko et al.: Limited Assessment of Respiratory Muscle Response to Nusinersen Treatment in Infants with Spinal Muscular Atrophy
por: LoMauro, Antonella, et al.
Publicado: (2020)

Limited Assessment of Respiratory Muscle Response to Nusinersen Treatment in Infants with Spinal Muscular Atrophy
por: Chacko, Archana, et al.
Publicado: (2020)

Ion Channel Gene Mutations Causing Skeletal Muscle Disorders: Pathomechanisms and Opportunities for Therapy
por: Maggi, Lorenzo, et al.
Publicado: (2021)

Cannot write session to /tmp/vufind_sessions/sess_sc2f5i7g6rk0jtbhtfhl8niced