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Muscle-directed Gene and Enzyme Replacement Therapies for Glycogen Storage Disorder Type II Pompe Disease

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Muscle-directed Gene and Enzyme Replacement Therapies for Glycogen Storage Disorder Type II Pompe Disease

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Myopathies, An Issue of Neurologic Clinics

Congential myopathies, Muscular dystropies, Glycogen storage diseases of muscle, and Idiopathic and Inflammatory myopathies are presented;

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Muscular Dystrophy

dystrophies and some novel treatments in glycogen storage disease by enzyme replacement are proposed. Both rehabilitation techniques and other;

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Gaucher Disease

research into this particular orphan disorder an invaluable prototype for the diagnosis, research, and treatment of others. Like many rare;

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Gaucher Disease

research into this particular orphan disorder an invaluable prototype for the diagnosis, research, and treatment of others. Like many rare;

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Fabry Disease

multi-system disorder, albeit with considerable phenotypic heterogeneity in onset and in severity; however, it is progressive, exhibits;

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Fabry Disease

multi-system disorder, albeit with considerable phenotypic heterogeneity in onset and in severity; however, it is progressive, exhibits;

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Advances in Diagnosis & Management of Glycogenosis II

years. To a large extent, this renewed interest is due to recent advances in diagnosing and treating the Lysosomal disease named Glycogenosis II;

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Advances in Diagnosis & Management of Glycogenosis II

years. To a large extent, this renewed interest is due to recent advances in diagnosing and treating the Lysosomal disease named Glycogenosis II;

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Regenerative Medicine for Degenerative Muscle Diseases

congenital myopathy, and new stem-cell based therapies and gene replacement therapy. Twelve expertly-authored chapters navigate the nuances;

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The Carbonic Anhydrases

of evolution of gene families and for site-directed mutagenesis in structure/function relationships, for protein folding and for transgenic and gene;

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Glycogen Synthase Kinase 3 (Gsk-3) And Its Inhibitors

Many researchers believe that GSK-3 and its inhibitors could lead to effective treatments for neurogenerative disorders, type II diabetes;

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Neurometabolic Hereditary Diseases of Adults

diagnosis. Information regarding how to deal with diseases with special therapy is provided (i.e. enzymatic replacement therapy in Fabry disease and;

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Gaucher's Disease

abnormalities of the lungs. GD is classified into three types: type 1 GD (GD1) is a chronic and non-neuronopathic accounting for 95% of GD cases, and;

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Samuels's Manual of Neurologic Therapeutics

of neuromuscular disorders using IV immunoglobulin; enzyme replacement therapy for genetic muscle disease; new information on the interventional treatment;

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Biopsy pathology of muscle

in clinical practice, both for diagnosis and for assessing progress in repeated biopsies during the course of a disorder and its treatment. The full;

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Muscle Gene Therapy

examines basic protocols for optimizing the muscle gene expression cassette and for evaluating the therapeutic outcomes, new developments in muscle;

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Duchenne Muscular Dystrophy 4 E

Duchenne Muscular Dystrophy, an inherited and progressive muscle wasting disease, is one of the most common single gene disorders found;

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Kidney Diseases and Renal Replacement Therapies, An Issue of Veterinary Clinics: Small Animal Practice

Guest editors Mark Acierno and Mary Labato highlight important areas in kidney failure and renal replacement therapies for all small;

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DNA Methylation and Complex Human Disease

, tailored epigenetic therapies, and the future uses of high-throughput methylome technologies. This volume includes many pertinent;

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Alpha-1-antitrypsin Deficiency

Alpha-1-antitrypsin Deficiency: Biology, Diagnosis, Clinical Significance, and Emerging Therapies is the authoritative reference on AATD;

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Phytochemicals

Understanding phytochemical-gene interactions provides the basis for individualized therapies to promote health as well as prevent and;

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Phytochemicals

Understanding phytochemical-gene interactions provides the basis for individualized therapies to promote health as well as prevent and;

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Neurochemistry of Metabolic Diseases

Metabolic disorder caused by altered levels of metabolism resulting pathophysiological abnormalities often leads to childhood death;

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Enzymology of Complex Alpha-Glucans

Glycogen and Starch: So Similar, yet so Different. Both carbohydrates are central to the primary metabolism of a large part of the living;

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Chronic Kidney Disease in Disadvantaged Populations

disparities in access to screening, prevention strategies, treatment protocols and renal replacement therapies. Chronic Kidney Disease;

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