Small Molecule Therapy for Genetic Disease

Herausgeber: Thoene, Jess G.

• Gebundenes Buch

Produktbeschreibung

Thoene focuses on innovative and progressive small molecule therapies with potentially life-saving therapeutic responses for metabolic genetic disorders.

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Produktdetails

• Verlag: Cambridge University Press
• Seitenzahl: 242
• Erscheinungstermin: 21. Oktober 2010
• Englisch
• Abmessung: 260mm x 183mm x 18mm
• Gewicht: 658g
• ISBN-13: 9780521517812
• ISBN-10: 0521517818
• Artikelnr.: 29934179

Inhaltsangabe

1. FDA and the regulation of small molecules for orphan diseases Marlene
Haffner and Tan Nguyen; 2. The office of rare diseases research: serving a
coordinating function at the national institutes of health Stephen C.
Groft; 3. Introduction to pharmacokinetics and pharmacodynamics Juan J. L.
Lertora and Konstantina M. Vanevski; 4. Biotin and biotin-responsive
disorders Barry Wolf and Kirit Pindolia; 5. Cobalamin treatment of
methylmalonic acidemias Hans C. Andersson; 6. Sapropterin treatment of
phenylketonuria Barbara K. Burton; 7. L-carnitine therapy in primary and
secondary carnitine deficiency disorders Susan C. Winter, Brian Schreiber
and Neil R. M. Buist; 8. Cysteamine treatment of nephropathic cystinosis
Jess Thoene; 9. Nitisinone use in hereditary tyrosinemia and alkaptonuria
William A. Gahl, Wendy J. Introne and Kevin O'Brien; 10. Alternative waste
nitrogen disposal agents for urea cycle disorders Gregory M. Enns; 11. PDMP
based glucosylceramide synthesis inhibitors for Gaucher and Fabry disease
James A. Shayman; 12. Betaine treatment for the homocystinurias Harvey L.
Levy and Amy Lawson-Yuen; 13. Zinc and tetrathiomolybdate for the treatment
of Wilson's disease George J. Brewer; 14. Small copper complexes for
treatment of ATP7A-related disorders Stephen G. Kaler.