Pharmacological chaperone therapy for lysosomal storage diseases

Future Medicinal Chemistry

Volumn 6, Issue 9, 2014, Pages 1031-1045

  Parenti, Giancarlo ^a,b   Moracci, Marco ^c   Fecarotta, Simona ^b   Andria, Generoso ^b  

^a TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^b UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^c CNR   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

1 DEOXYNOJIRIMYCIN; CHAPERONE; MIGALASTAT; MOLECULAR LIBRARY;

BINDING SITE; BIOCHEMISTRY; CONFORMATION; CONFORMATIONAL TRANSITION; DRUG BIOAVAILABILITY; DRUG EFFICACY; DRUG IDENTIFICATION; ENZYME REPLACEMENT; FABRY DISEASE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; TRANSLATIONAL RESEARCH; ADMINISTRATION AND DOSAGE; ALLOSTERISM; CHEMICAL STRUCTURE; CHEMISTRY; DRUG EFFECTS; ENZYMOLOGY; LYSOSOMAL STORAGE DISEASES; METABOLISM; MOLECULAR LIBRARY; PHARMACOLOGY; THERAPEUTIC USE;

ALLOSTERIC REGULATION; HUMANS; LYSOSOMAL STORAGE DISEASES; MODELS, MOLECULAR; SMALL MOLECULE LIBRARIES;

EID: 84905164552     PISSN: 17568919     EISSN: 17568927     Source Type: Journal    
DOI: 10.4155/fmc.14.40     Document Type: Review

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