Therapeutics development for cystic fibrosis: A successful model for a multisystem genetic disease

Annual Review of Medicine

Volumn 62, Issue , 2011, Pages 107-125

  Ashlock, Melissa A ^a   Olson, Eric R ^b  

^a CYSTIC FIBROSIS FOUNDATION   (United States)

^b VERTEX PHARMACEUTICALS   (United States)

Author keywords

basic defect; CFTR modulator; drug discovery; patient registry; symptomatic therapy

Indexed keywords

AGENTS AFFECTING PROTEIN METABOLISM; ATALUREN; BICARBONATE; CHLORIDE; DENUFOSOL; EPITHELIAL SODIUM CHANNEL; LANCOVUTIDE; MANNITOL; SODIUM; SODIUM CHLORIDE; TRANSMEMBRANE CONDUCTANCE REGULATOR; TRYPSINOGEN; UNCLASSIFIED DRUG; VX 770; VX 809; XYLITOL;

ARTICLE; CLINICAL FEATURE; CYSTIC FIBROSIS; DRUG MECHANISM; DRUG SAFETY; DRUG TOLERABILITY; FORCED EXPIRATORY VOLUME; GENE MUTATION; GENE THERAPY; GENOTYPE PHENOTYPE CORRELATION; HUMAN; MORTALITY; NEWBORN SCREENING; NONHUMAN; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RESPIRATORY FAILURE; RISK FACTOR; SWEAT TEST; TREATMENT DURATION;

CLINICAL TRIALS AS TOPIC; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG DISCOVERY; DRUGS, INVESTIGATIONAL; FEMALE; GENISTEIN; HUMANS; MALE; MUTATION; PHENYLBUTYRATES; PROTEIN KINASE INHIBITORS; PSEUDOMONAS AERUGINOSA; PSEUDOMONAS INFECTIONS; PULMONARY DISEASE, CHRONIC OBSTRUCTIVE; RISK FACTORS;

EID: 79551596804     PISSN: 00664219     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev-med-061509-131034     Document Type: Article

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