Clinical Trials to Date
Abstract Diseases that afflict the nervous system remain among the most difficult pathologies for clinicians to manage. Currently there are nearly 1000 clinical trials that have been launched worldwide to…
Gene Therapy for Amyotrophic Lateral Sclerosis: Therapeutic Transgenes
Abstract Amyotrophic lateral sclerosis (ALS) is a disease with a poorly understood etiology. There are a wide variety of abnormalities present, including mitochondrial dysfunction, glutamate excitotoxicity, oxidative stress, astrogliosis and…
Introduction to Gene and Stem-Cell Therapy
Abstract Motor neuron diseases, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), are devastating neurodegenerative diseases that are progressive and fatal. There is no cure and the…
Molecular and Extracellular Cues in Motor Neuron Specification and Differentiation
Abstract Motor neurons (MNs) are a diverse group of cells without which life would not be possible. MNs are responsible for integrating signals from the brain and the sensory systems…
Molecular Mechanisms of Amyotrophic Lateral Sclerosis
Abstract Amyotrophic lateral sclerosis (ALS) is a progressive, fatal, neurodegenerative disorder whose pathological hallmark is the loss of motor neurons of the corticospinal pathway. While ALS is a heterogeneous disorder,…
Genetics of Spinal Muscular Atrophy
Abstract Spinal muscular atrophy (SMA) is a group of disorders defined by specific lower motor neuron involvement. Proximal 5q SMA, the most common of these disorders, is caused by loss…
An Introduction to the Natural History, Genetic Mapping, and Clinical Spectrum of Spinal Muscular Atrophy
Abstract Spinal muscular atrophy is a genetic neurodegenerative disorder affecting muscle tone and function, primarily in children. Disease onset varies from in utero to adult, and its progression and severity…
Genetics of Amyotrophic Lateral Sclerosis
Abstract The identification of disease-causing mutations has proven to be essential for our understanding of the pathogenesis and progression of human disorders. The identification of such genes can lead to…
Natural History of Amyotrophic Lateral Sclerosis
Abstract Amyotrophic lateral sclerosis (ALS) is the most common adult-onset motor neuron disease. ALS was first described in the medical literature in the mid-19th century, but despite its early recognition…
Transcranial Magnetic Stimulation
Abstract More than 30 years have already passed since the seminal article of Baker et al., in which a transcranial magnetic stimulation (TMS) pulse over the motor cortex (MC) was successfully…
