Gene Therapy for Spinal Muscular Atrophy

Sep 9, 2018 by in NEUROLOGY Comments Off on Gene Therapy for Spinal Muscular Atrophy

Abstract Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease that impacts approximately 1:10,000 live births. SMA is caused by deletion or loss-of-function mutations in the Survival Motor Neuron 1…

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Stem Cell Therapy for Amyotrophic Lateral Sclerosis

Sep 9, 2018 by in NEUROLOGY Comments Off on Stem Cell Therapy for Amyotrophic Lateral Sclerosis

Abstract Amyotrophic lateral sclerosis (ALS) is a challenging disease characterized by progressive loss of motor neurons. This is manifested in deterioration of strength, gait, speech, swallowing, respiratory function, and eventually…

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Clinical Trials to Date

Sep 9, 2018 by in NEUROLOGY Comments Off on 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…

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Introduction to Gene and Stem-Cell Therapy

Sep 9, 2018 by in NEUROLOGY Comments Off on 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…

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Molecular Mechanisms of Amyotrophic Lateral Sclerosis

Sep 9, 2018 by in NEUROLOGY Comments Off on 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,…

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Genetics of Spinal Muscular Atrophy

Sep 9, 2018 by in NEUROLOGY Comments Off on 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…

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