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 73 clinical trials
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 …
Clinical Trial of Mexiletine Hydrochloride for Spinal and Bulbar Muscular Atrophy
The purpose of this clinical trial is to evaluate the efficacy and safety of mexiletine hydrochloride in patients with spinal and bulbar muscular atrophy. The main questions it aims to answer are: Does mexiletine hydrochloride improve the ALSFRS-R score in spinal and bulbar muscular atrophy patients? Participants will: Take mexiletine …
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with spinal muscular atrophy, duchenne muscular dystrophy, or cerebral palsy.
Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
In recent years, knowledge of neuromuscular diseases has advanced considerably, and new therapeutic avenues are beginning to emerge. The proliferation of clinical trials has created a need to identify biomarkers that are both sensitive to changes and specific to the disease. Current gait tests only consider the time factor and …
Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS), is a rapidly progressive neurodegenerative disorder, usually leading to death from respiratory failure in 3-5 years. Riluzole, the only drug currently available, only modestly prolongs survival and does not improve muscle strength or function. In ALS, loss of functional motor neurons is initially compensated for by …
Repetitive Transorbital Alternating Current Stimulation for Optic Neuropathies
The purpose of this study is to test the efficacy and feasibility of an intervention protocol for home-based repetitive transorbital alternating current stimulation (rtACS) for the treatment of visual impairment in people with optic neuropathy. The primary aims are to evaluate the effectiveness of home-based rtACS to ameliorate the progressive …
Hyperbaric Oxygen Therapy for Optic Neuropathies
The purpose of the study is to evaluate the neuroprotective efficacy of hyperbaric oxygen for the treatment in patients with optic neuropathy.
Electrical Stimulation for the Treatment of Optic Neuropathies
The overall aim of this study is to see whether long-term electrical stimulation with a home-stimulation device works well and is safe for the treatment of open-angle glaucoma. Open-Angle Glaucoma is a disease where the nerves in the back of your eye die off faster than expected regardless of your …
The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
The goal of this clinical trial is to investigate the effect of a muscle-mimicking, fabric-type shoulder orthosis on functional movements of the upper limb in patients with neuromuscular disorder. The main questions it aims to answer are: What is the impact of the muscle-mimicking, fabric-type shoulder orthosis on upper limb …
Stem Cell Ophthalmology Treatment Study II
This study will evaluate the use of autologous bone marrow derived stem cells (BMSC) for the treatment of retinal and optic nerve damage or disease.