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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.

RESULTS

Found (131) clinical trials

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R. The ...

Phase

The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Phase

Blood-Brain Barrier Opening Using MR-Guided Focused Ultrasound in Patients With Amyotrophic Lateral Sclerosis

The purpose of this study is to evaluate the safety, tolerability, and feasibility of Blood-Brain Barrier (BBB) opening using transcranial MRI-guided focused ultrasound in conjunction with an intravenous ultrasound contrast agent in patients with Amyotrophic Lateral Sclerosis (ALS).

Phase N/A

Biomarkers of Synaptic Damage in Multiple Sclerosis

Blood and cerebrospinal fluid samples will be subjected to the procedures required for the isolation of the different components immediately after the withdrawal. Subsequently, the levels of microRNAs, cytokines, chemokines, cell growth factors, neuronal damage markers (tau, phosphorylated and truncated tau, neurofilaments) and mitochondrial (lactate) and free d-amino acids (Objective ...

Phase N/A

Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)

To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)

Phase

Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL)

Frontotemporal Lobar Degeneration (FTLD) is the neuropathological term for a collection of rare neurodegenerative diseases that correspond to four main overlapping clinical syndromes: frontotemporal dementia (FTD), primary progressive aphasia (PPA), corticobasal degeneration syndrome (CBS) and progressive supranuclear palsy syndrome (PSPS). The goal of this study is to build a FTLD ...

Phase N/A

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

Spinal Muscular Atrophy (SMA) is a progressive, recessively-inherited neuromuscular disease characterized by weakness and muscle atrophy due to the loss of spinal cord motor neurons. The results from this study would provide preliminary data, using non-invasive methods, on oxidative capacity in ambulatory SMA patients and disease controls to aid in ...

Phase N/A

Trial Evaluating the Interest of Noninvasive Ventilation in NAVA Mode in Respiratory Decompensations Children With Infantile Spinal Muscular Atrophy Type II

This is a prospective, interventional, sequential, open-label research involving ASI II children with acute respiratory decompensation requiring ventilatory support by NIV. Airway pressure, transcutaneous CO2 monitoring and hemodynamic parameters will be recorded continuously during the study, after inclusion of the patient.

Phase N/A

Study of Predictive Factors of Progression of Lateral Amyotrophic Sclerosis

This is a prospective observational multicentric French study of a cohort of 1000 ALS patients, 100 neurological controls and 200 healthy controls followed from the first signs to the end of the disease. The aim of the present study is therefore to determine the clinical, biological, imaging, and electrophysiological biomarkers ...

Phase N/A

A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells in Patients With Amyotrophic Lateral Sclerosis (ALS)

This is a study of transplantation of Astrocytes derived from human embryonic stem cells, in patients with Amyotrophic Lateral Sclerosis (ALS). There will be no change in the routine ALS treatment of the patients enrolled into the study. Treatment will be administered in addition to the appropriate standard of care ...

Phase