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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 (18) clinical trials

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

The goal of the BrainGate2 research and development project is to identify the core methods and features for a medical device that could allow people with paralysis to recover a host of abilities that normally rely on the hands.

Phase N/A

Genetics of Familial and Sporadic ALS

The investigators long term goals are to improve diagnosis and develop effective treatments that arrest or ameliorate symptoms of ALS, and possibly delay or prevent disease onset in individuals at risk for developing familial ALS (FALS). In order to do this one must understand how disease develops at a molecular ...

Phase N/A

Derivation of Induced Pluripotent Stem Cells From an Existing Collection of Human Somatic Cells

The potential to reprogram somatic cells into an embryonic state raises multiple basic research questions related both to the process of reprogramming and the properties of iPS cells. We will use various approaches to study the molecular mechanisms and processes that occur during reprogramming. We will use various experimental systems ...

Phase N/A

Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene

Spinal Muscular Atrophy (SMA) is a neuromuscular disorder characterized by loss of motor neurons in the anterior horn of the spinal cord and leading to muscle atrophy. SMA has an autosomal recessive inheritance and affects 1 in 6000 infants with a carrier frequency of 1 in 40. In most cases, ...

Phase N/A

Oral Nutritional Supplementation in Amyotrophic Lateral Sclerosis (ALS) Patients

Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease with a median age at time of diagnosis of 65 years. In France, the incidence ranges between 1.5 and 2.5/100 000 person-year of follow-up. The disease is related to progressive degeneration of motor neurons in the two voluntary motor pathways. It ...

Phase N/A

Effect of Noninvasive Ventilation on Lung Function in Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS), or "Lou Gehrig's Disease", is a fatal disorder that causes progressive degeneration and weakening of the muscles of breathing, leading to breathing insufficiency and eventually breathing failure. This breathing insufficiency is commonly treated with a breathing assistance device, known as noninvasive positive pressure ventilation (NIPPV). While ...

Phase N/A

Olanzapine for the Treatment of Appetite Loss in Amyotrophic Lateral Sclerosis (ALS)

After randomization, there is a placebo-controlled parallel group treatment with 10 mg OLN in combination with the standard treatment of Riluzole (100mg/day)(Group 1) in comparison to treatment with placebo in combination with 100 mg RIL (Group 2). Study drug will be provided as 5 mg tablets. OLN will be begun ...

Phase

Mitochondrial Functions and Oxidative Stress in ALS Patients

In Amyotrophic Lateral Sclerosis (ALS), malnutrition is frequent (16 to 50 % of the patients) and is an independent prognostic factor. One of the implicated factors is the increase of resting energy expenditure (REE) which can be found in about 50 % of ALS patients. The origin of this hypermetabolism ...

Phase N/A

Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis

Background Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of ...

Phase

Molecular Imaging Modality by Positron Emission Tomography Using 18F-X : Study of Microglial Activation in Amyotrophic Lateral Sclerosis

18F-X PET will be carried out requiring arterial sampling in 2 patients suffering from ALS and 2 normal subjects in order to evaluate the 18F-X quantification. Then simplified PET using 18F-X will be carried out in 13 patients and 13 normal subjects. Binding potential maps showing specific binding of 18f-X ...

Phase