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Found 9 clinical trials
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA, as measured by change in Children's

gene deletion
nusinersen
muscle atrophy
  • 42 views
  • 22 Sep, 2021
  • 55 locations
A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec

The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received

gene deletion
nusinersen
  • 0 views
  • 12 Aug, 2021
  • 13 locations
Ultrasound-assisted vs Landmark Based Intrathecal Administration of Nusinersen

Intrathecal administration of Nusinersen, an antisense oligonucleotide capable of increasing Survival Motor Neuron protein production, has been tested in Spinal Muscular Atrophy (SMA) to improve

  • 0 views
  • 18 Feb, 2021
  • 1 location
Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)

This study aims to refine the capability of Multispectral Optoacoustic Tomography (MSOT) and Magnet Resonance Imaging (MRI) to characterise the molecular composition of muscle tissue non-invasively and to evaluate the therapeutic response in patients with spinal muscular atrophy (SMA) over time.

spinraza
optoacoustic tomography
spinal muscular atrophy type i
muscle atrophy
  • 0 views
  • 23 Jan, 2021
  • 1 location
Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients

The "SMOB" project intends to contribute to fill the gap with reliable and operational outcome measures for type III and IV SMA. In analysing the reliability in imaging (spinal and muscular), electrophysiology analysis (MUNIX), and evaluate the evolution of respiratory function for 50 patients' cohort. The investigators would also take …

atrophy
  • 0 views
  • 12 Sep, 2021
  • 10 locations
Observational Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

The primary objective is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in Korea. The secondary objective is to evaluate the effectiveness of nusinersen

spinraza
nusinersen
  • 2 views
  • 04 Apr, 2021
  • 14 locations
Non-Interventional Postmarketing Surveillance Study of Nusinersen Sodium Injection

The primary objective of this study is to evaluate the safety of nusinersen sodium injection in the postmarketing setting in China.The secondary objectives are to collect data on the efficacy

nusinersen
  • 0 views
  • 31 May, 2021
  • 9 locations
Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen

Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.

  • 0 views
  • 19 Feb, 2021
  • 8 locations
Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)

The primary objective of this research protocol is to study and follow the course of motor neuron loss in individuals with spinal muscular atrophy (SMA) using the electrophysiological technique of motor unit number estimation (MUNE). This study is based on the hypothesis that the electrophysiological technique of motor unit number …

muscle atrophy
  • 0 views
  • 25 Jan, 2021
  • 1 location