spinal-cord-disorders Clinical Trials
A listing of spinal-cord-disorders medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 587 clinical trials
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
This is an observational study to investigate the improvement of NMJ defects in adult patients with SMA following treatment with Risdiplam. Eligible patients will have received treatment with daily oral Risdiplam after receiving approval through their commercial insurance or drug assistance program. All subjects will be evaluated at one visit. …
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is caused by the homozygous loss of the Survival Motor Neuron (SMN) 1 gene, which leads to degeneration of spinal alpha-motor neurons and muscle atrophy. Three treatments have been approved for SMA but the available data show interpatient variability in therapy response and, to date, individual …
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
This is an observational multicenter retrospective and prospective study on natural history of SMA also considering the 'new natural history' secondary to the availability of commercially available therapies. All the patients enrolled to date in the Italian registry, if not part of clinical trials, will be included in the present …
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
The purpose of this project is to investigate the utility of the SMA EFFORT, an SMA-specific patient-reported outcome measure, to assess perceived physical fatigability that is anchored to intensity and duration of activities. We aim to characterize perceived physical fatigability (PPF) in a diverse cohort of people with SMA (pwSMA) …
A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022
Spinal Muscular Atrophy (SMA) is a rare neuromuscular condition, characterised by loss of motor neurons as a result of a mutation in the survival motor neuron gene. This results in muscle wasting and in the most common and severe type, death before 24 months. Over the recent years there has …
Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
In this observational study, researchers are looking at the effects of spinal muscular atrophy (SMA) drugs on the muscles and nerve cells in patients with SMA. Primary Objectives To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA. To evaluate patients with …
Evaluate the Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
Evaluate the diagnostic journey, patient experience, and disparities in the treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System.
Genotype-phenotype Correlation and Pathogenic Mechanism in Hereditary Ataxia
The investigators aimed to find appropriate biomarkers such as serum neurofilament light chain in reflecting disease severity in hereditary ataxia from a large cohort during long-term follow-up. The disease severity is indicated by clinical scales and brain MRI tests.
Neuroimaging Changes in Hereditary Ataxia
This study aim to investigate the neuroimaging changes of hereditary ataxia patients, especially in the SCA3 patients in preclinical or mild stage.
En Bloc Resection Versus Separation Surgery Combined With Radiotherapy for the Treatment of Spinal Oligometastatic Tumor.
The purpose of this clinical trial is to explore the impact of En bloc surgery and separation surgery combined with radiation therapy on the prognosis and survival of patients with spinal oligometastatic cancer, describe the clinical results, and optimize future treatment goals
