Found 6,933 clinical trials
A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
In this study, researchers will learn more about the safety of tofersen, also known as Qalsody®. This is a drug available for doctors to prescribe for participant with a certain type of amyotrophic lateral sclerosis, also known as ALS. This type is in participant who have a mutation in the …
Disease Biosignatures in ALS/FTD Spectrum: New Impactful Biological Perspectives Beyond Clinical Approaches
Diagnosis of ALS/FTD disease spectrum is challenging because it largely relies on clinical symptoms. Identifying novel biomarkers is essential for a paradigm shift towards a more precise biological-based diagnosis. To achieve this aim, having access to proper specimens and analytical methods is crucial. Our team of experts in neurology, biology, …
Electrophysiology and Ultrasound of Respiratory Muscles and Respective Nerves to Predict Respiratory Insufficiency in ALS
Respiratory insufficiency is a common complication in amyotrophic lateral sclerosis (ALS). Survival can be prolonged with noninvasive ventilation (NIV). The goal of this study is to assess the value of electromyography of respiratory muscles as well as the ultrasound of the diaphragm and nerves involved in respiration in predicting the …
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs). There is a clear medical unmet need to provide a battery of appropriate locomotor gait assessments to determine the risk of falling for patients with …
A US Study That Observes How Parkinson's Disease Changes Over Time in Patients Who Still Have Movement Symptoms Despite Taking Parkinson's Medications
This is an observational study in which data are collected and studied from Parkinson's disease patients who have movement symptoms despite taking standard Parkinson's medications. In observational studies, observations are made without any changes to the participant's healthcare or treatment plan. No investigational product will be administered in this study, …
Italian Translation and Validation of the GIDS-PD Scale for Parkinson's Disease
This study aims to translate and validate the Gastrointestinal Dysfunction Scale for Parkinson's Disease (GIDS-PD) into Italian to enable the assessment of gastrointestinal symptoms in Italian-speaking patients with Parkinson's disease. The psychometric properties of the translated instrument will be evaluated through a multi-phase process involving translation/back-translation, pre-testing, and large-scale validation …
Dynamical Electrophysiology and Neuropathology in Lewy's Bodies Disease
Lewy body disease (LBD) is a neurodegenerative disorder that causes cognitive and behavioral problems. However, it is difficult to diagnose, and its pathophysiology is still not fully understood. The usual diagnostic criteria for LBD have excellent specificity but poor sensitivity. In particular, we lack paraclinical tests that can make a …
Pairing Subjective Patient Rating and DBS Programming
This multicenter, prospective and retrospective diagnostic study investigates personalized programming strategies for deep brain stimulation (DBS) in patients with Parkinson's disease. DBS of the subthalamic nucleus (STN) is an established therapy for advanced Parkinson's disease; however, optimization of stimulation parameters remains time-consuming and resource-intensive due to the growing complexity of …
Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease
The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged duration)
Predictors of Clinical Outcomes of Deep Brain Stimulation in Parkinson's Disease
The goal of this observational study is to identify factors in blood that are associated with response to deep brain stimulation (DBS) surgery in patients with Parkinson's disease. The main questions it aims to answer are:What factors in blood (proteins and RNA) are associated with good vs poor response to …
