Pharmacological chaperone therapy: Preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders

Molecular Therapy

Volumn 23, Issue 7, 2015, Pages 1138-1148

  Parenti, Giancarlo ^a,b   Andria, Generoso ^a   Valenzano, Kenneth J ^c  

^a UNIVERSITY OF NAPLES FEDERICO II   (Italy)

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

^c Amicus Therapeutics   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1 DEOXYNOJIRIMYCIN; AFEGOSTAT; AGALSIDASE ALFA; AGALSIDASE BETA; ALPHA GLUCOSIDASE INHIBITOR; AMBROXOL; GALACTOSE; GLUCOSAMINE; LARAZOTIDE; MEXAZOLAM; MIGALASTAT; MIGLUSTAT; N ACETYLGLUCOSAMINE THIAZOLINE; N OCTYL 4 EPI BETA VALIENAMINE; OT 1001; PYRIMETHAMINE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; UNCLASSIFIED DRUG; CHAPERONE; LIGAND;

ATAXIA; BIOACCUMULATION; CELL MIGRATION; COORDINATION DISORDER; DISEASE COURSE; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; ENDOPLASMIC RETICULUM; ENZYME ACTIVITY; ENZYME REPLACEMENT; ENZYME SYNTHESIS; FABRY DISEASE; GAA GENE; GAUCHER DISEASE; GBA1 GENE; GENE; GLYCOGEN STORAGE DISEASE TYPE 2; GM1 GANGLIOSIDOSIS; GM2 GANGLIOSIDOSIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IN VITRO STUDY; IN VIVO STUDY; LYSOSOME STORAGE DISEASE; MISSENSE MUTATION; MUSCLE WEAKNESS; NEURONAL CEROID LIPOFUSCINOSIS; NONHUMAN; PHARMACOLOGICAL CHAPERONE THERAPY; PHARMACOLOGICAL PROCEDURES; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHENOTYPE; PILOT STUDY; PREVALENCE; PROTEIN FOLDING; PROTEIN HOMEOSTASIS; PROTEIN SYNTHESIS; QUALITY CONTROL; QUALITY OF LIFE; REVIEW; SANFILIPPO SYNDROME; SEIZURE; SINGLE DRUG DOSE; THERMODYNAMICS; DRUG EFFECTS; ENZYME STABILITY; GENETICS; LYSOSOMAL STORAGE DISEASES; LYSOSOME; PATHOLOGY; PROTEIN DEGRADATION;

ENZYME STABILITY; HUMANS; LIGANDS; LYSOSOMAL STORAGE DISEASES; LYSOSOMES; MOLECULAR CHAPERONES; PROTEIN BIOSYNTHESIS; PROTEIN FOLDING; PROTEOLYSIS;

EID: 84934437646     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.62     Document Type: Review

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