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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.


Found (124) clinical trials

National Registry for Egyptian Pediatric Neuromuscular Diseases

Aims: An open-ended multi-center, national Egyptian study to collect and analyze data for children with Neuromuscular Diseases (NMD) inherited NMD (spinal muscular atrophy (SMA), Duchenne/Becker and congenital muscular dystrophies (DMD/BMD, CMD), congenital myopathies, and congenital myasthenic syndromes) and acquired NMD (neuropathies, myasthenia gravis and myositis). Participants: Eligible infants and children ...

Phase N/A

Natural History of Types 2 and 3 SMA in Taiwan

As with other rare diseases, individual groups of SMA have therefore opted to share patient information in the form of clinical sites to increase the overall patient cohorts on which clinical outcomes and new assisted-healthcare technologies can be assessed. Using the collaborative and retrospective study of types 2 and 3 ...

Phase N/A

Safety and Tolerability of Perampanel in Amyotrophic Lateral Sclerosis Patients

Amyotrophic lateral sclerosis (ALS), the most common motor neuron disease, is a fatal progressive neurodegenerative disease affecting motor cortex, brainstem and spinal cord leading to motor neuron death. It is a devastating disease of the anterior and lateral corticospinal tracts with approximately 3 years mean duration from symptoms onset to ...


Intraspinal Transplantation of Autologous ADRC in ALS Patients

Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. ...


Spinal Interneuron Excitability in ALS

Given the all-or-none properties of neuromuscular junctions, the electromyogram (EMG) reflects the activity of spinal motoneurons. These neurons are the last order neurons in all neural pathways involved in motor control. Modification of the afferent neural activity, whatever its level (spinal or supraspinal), will affect the excitability of spinal motoneurons ...

Phase N/A

Rapamycin Treatment for ALS

This is a phase II randomized, double-blind, placebo-controlled, multicenter clinical trial for people with ALS. The aim is to study the biological and clinical effect of Rapamycin (in two different doses) in addition to Riluzole on ALS patients through comparison with patients treated with Riluzole and placebo. Rapamycin has been ...


Predict to Prevent Frontotemporal Lobar Degeneration (FDT) and Amyotrophic Lateral Sclerosis (ALS)

This study will investigate whether cognitive deficits, structural and functional changes can be detected before symptom onset in presymptomatic mutation carrier. The main objectives of the project are to identify novel cognitive, brain imaging markers and peripheral biomarkers for early diagnosis of FTLD, and to follow disease progression. Methodology Recruitment ...

Phase N/A

Trial of Amivita in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder affecting upper and lower motor neurons. Survival is typically 2 to 5 years from symptom onset; death is usually from respiratory paralysis. Standard therapy is with Riluzole 100 mg/day, a FDA approved treatment for ALS that has a small effect on ...

Phase N/A

Noninvasive Assessment of Neuronal Damage by MRI Sodium ( 23Na ) in Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease affecting the central and peripheral motor neurons, characterized by the rapidity of its evolution (median survival of 3 years). The pathophysiology of the disease is still poorly understood. Neuronal death results from several cellular mechanisms entangled, including mitochondrial dysfunction. The absence of ...

Phase N/A

ALS Treatment Extension Study

Treatment extension study for ALS/MND patients who participated in phase 1 study CMD-2016-001, completed assessments following six 28-day cycles of treatment, and whom the Investigator considers would benefit from continued CuATSM treatment. The same safety assessments as in the CMD-2016-001 study will be conducted after each cycle of treatment and ...