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Found 34 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 spinal muscular atrophy (SMA), as measured

gene deletion
nusinersen
muscle atrophy
  • 144 views
  • 22 Dec, 2021
  • 64 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
  • 15 Dec, 2021
  • 20 locations
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene ≤ 24 months and weighing ≤ 17 kg, over a 18-month period post infusion.

gene mutation analysis
synagis
palivizumab
  • 0 views
  • 26 Dec, 2021
  • 1 location
Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival

a 12
  • 0 views
  • 02 Jan, 2022
  • 9 locations
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
Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)

-invasively and to evaluate the therapeutic response in patients with spinal muscular atrophy (SMA) over time.

spinal muscular atrophy type i
spinraza
optoacoustic tomography
muscle atrophy
  • 0 views
  • 23 Jan, 2021
  • 1 location
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
Intranasal Neurostimulation in Ameliorating Symptoms of Neuropathic Corneal Pain

This study is a prospective, interventional, open-label, single arm, non-randomized trial treating 30 patients with peripheral or mixed neuropathic corneal pain at their baseline exam and following 45 days and 90 days of daily use with the TrueTear ITN device.

  • 7 views
  • 14 Mar, 2021
  • 1 location
A Phase I Feasibility Study of an Intracortical Visual Prosthesis (ICVP) for People With Blindness

The purpose of this study is to determine the feasibility of producing artificial vision in persons with blindness. Study participants will have wireless electrical stimulators implanted into the cortical vision processing areas of their brains. The ability of the participants to perceive artificial vision in response to electrical stimulation will …

  • 0 views
  • 21 Jan, 2022
  • 1 location
Pediatric Powered Wheelchair Standing Devices: An Exploratory Study

For children who use a power wheelchair, a powered wheelchair standing device (PWSD) may be considered for daily use. A PWSD allows a child to electronically move between sitting and standing and can be driven in either position. Existing published PWSD research in pediatrics is limited to boys with Duchenne …

muscular dystrophy
  • 0 views
  • 20 Nov, 2021
  • 1 location