Cargando…

Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 – A Prospective Observational Study

OBJECTIVE: Spinal muscular atrophy (SMA) is a progressive autosomal recessive motor neuron disease caused by loss of the SMN1 gene. Based on randomized clinical trials in children with SMA type 1 and 2, Nusinersen has been approved as the first treatment for all types of SMA, including adults with S...

Descripción completa

Detalles Bibliográficos
Autores principales:	Walter, Maggie C., Wenninger, Stephan, Thiele, Simone, Stauber, Julia, Hiebeler, Miriam, Greckl, Eva, Stahl, Kristina, Pechmann, Astrid, Lochmüller, Hanns, Kirschner, Janbernd, Schoser, Benedikt
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2019
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6918909/ https://www.ncbi.nlm.nih.gov/pubmed/31594243 http://dx.doi.org/10.3233/JND-190416

Ejemplares similares

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)

Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study
por: Kiefer, Petra, et al.
Publicado: (2020)

De-duplicating patient records from three independent data sources reveals the incidence of rare neuromuscular disorders in Germany
por: König, Kirsten, et al.
Publicado: (2019)

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

Quality of Life in SMA Patients Under Treatment With Nusinersen
por: Mix, Lucas, 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)

A multi-source approach to determine SMA incidence and research ready population
por: Verhaart, Ingrid E. C., et al.
Publicado: (2017)

SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy
por: Pechmann, Astrid, et al.
Publicado: (2019)

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)

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

Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care
por: Schorling, David C., et al.
Publicado: (2020)

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)

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

Improvements in Walking Distance during Nusinersen Treatment – A Prospective 3-year SMArtCARE Registry Study
por: Pechmann, Astrid, et al.
Publicado: (2023)

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

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

Coagulation disorders in Duchenne muscular dystrophy? Results of a registry-based online survey
por: Schorling, David C., et al.
Publicado: (2020)

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)

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

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)

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis
por: Landfeldt, Erik, et al.
Publicado: (2020)

Mechanographic analysis of the timed 4 stair climb test – methodology and reference data of healthy children and adolescents
por: Schorling, David C., et al.
Publicado: (2023)

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)

Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review
por: Landfeldt, Erik, et al.
Publicado: (2019)

Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model
por: MacCannell, Drew, et al.
Publicado: (2022)

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

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)

Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
por: Landfeldt, Erik, et al.
Publicado: (2021)

Nociceptive pain in adult patients with 5q-spinal muscular atrophy type 3: a cross-sectional clinical study
por: Sagerer, Elena, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_e372dmovuckuiam6ffoevp1ss2