spinal-muscular-atrophy Clinical Trials
A listing of spinal-muscular-atrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 76 clinical trials
A Study of NIDO-361 in Patients With SBMA
The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).
Hippotherapy in Children With Spinal Muscular Atrophy
The randomized controlled trial is aimed to discover the physiotherapy and hippotherapy effect and efficacy on children with SMA. The concept is to utilized two types of physiotherapy - the first concept is classic physiotherapy and the second one is hippotherapy. The hippotherapy concept will be in intervals of 15 …
Spinal Muscular Atrophy on Wheels, Using Power Mobility
The group of children diagnosed with Spinal Muscular Atrophy (SMA) has serious restrictions on participation. SMA is a neuromuscular disease that leads to neuromusculoskeletal disorders that limit functional activities, sometimes making it impossible to sit down autonomously and to walk. Scientific evidence has highlighted the importance of implementing physiotherapy interventions …
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.
High-intensity Interval Training in Patients With Spinal Muscular Atrophy
Patients with spinal muscular atrophy who are wheelchair users often experience lower back - and gluteal pain, reduced sleep quality, constipation and reduced quality of life - symptoms that regular exercise could potentially alleviate. However, only very little research has been done on exercise for patients who are wheelchair users. …
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
There is no cure to arrest or delay SBMA progression. It is estimated that ~1000 individuals are affected by SBMA in Italy at any given time (prevalence: 1.5/100000) with an annual incidence of 0.19/100000 males. Here, we are going to test the potential of beta2-agonist stimulation on muscle as a …
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA). All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving …
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation. The main problems are posture disorders, scoliosis, pelvic curvature, contracture, hip dislocation, foot and chest deformities. In this study, examining the …
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 …