View clinical trials related to SMA II.
Filter by:The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 2 (SMA 2) patients.
This study aims to evaluate the safety, usability and acceptability of a motorized mobility assistance exoskeleton. The protocol explores the use of the motorized device during static and dynamic rehabilitation sessions focused on walking and functional activities, with the expectation of evaluating the safety and usability of the device in the population studied. The protocol has been focused on defining how the device can be used appropriately in this population in a safe and effective manner by rehabilitation specialists. The study also aims to assess the safety of clinical staff who implement the intervention.
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the Functional Ambulation Classification (FAC) as a consequence of their disease, as well as the assessment of other physical, functional and quality of life parameters of interest. For this purpose, the exoskeleton will be used as a rehabilitation and walking assistance tool. Before starting the study programme and if deemed necessary, a preliminary phase consisting of 1 to 3 sessions will be carried out during which the patient will be shown the exoskeleton for the first time and it will be considered whether any improvements to the different parts of the device or the software are necessary to increase the ergonomics and comfort of the patient.
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.