12825 results in Pharmacology
Frontmatter
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2 - The Office of Rare Diseases Research: Serving a coordinating function at the National Institutes of Health
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14 - Small copper complexes for treatment of acquired and inherited copper deficiency syndromes
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11 - PDMP-based glucosylceramide synthesis inhibitors for Gaucher and Fabry diseases
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3 - Introduction to pharmacokinetics and pharmacodynamics
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Small Molecule Therapy for Genetic Disease
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6 - Sapropterin treatment of phenylketonuria
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Contributors
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SECTION III - UTILIZATION OF ALTERNATIVE PATHWAYS
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12 - Betaine treatment for the homocystinurias
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7 - L-carnitine therapy in primary and secondary carnitine deficiency disorders
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10 - Alternative waste nitrogen disposal agents for urea cycle disorders
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9 - Nitisinone use in hereditary tyrosinemia and alkaptonuria
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Index
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SECTION I - INFRASTRUCTURE
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4 - Biotin and biotin-responsive disorders
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13 - Zinc and tetrathiomolybdate for the treatment of Wilson disease
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Contents
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1 - The U.S. Food and Drug Administration and the regulation of small molecules for orphan diseases
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Plates section
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