Novel personalized therapies for cystic fibrosis: Treating the basic defect in all patients

Journal of Internal Medicine

Volumn 277, Issue 2, 2015, Pages 155-166

  Amaral, M D ^a  

^a UNIVERSITY OF LISBON   (Portugal)

Author keywords

Monogenic disorder; Mutation specific therapies; Personalized therapy; Rare diseases

Indexed keywords

ANTIBIOTIC AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; MUCOLYTIC AGENT; AMINOGLYCOSIDE; AMINOPHENOL DERIVATIVE; ANTIINFECTIVE AGENT; ANTIINFLAMMATORY AGENT; ATALUREN; BIOLOGICAL MARKER; GENISTEIN; N-(2,4-DI-TERT-BUTYL-5-HYDROXYPHENYL)-4-OXO-1,4-DIHYDROQUINOLINE-3-CARBOXAMIDE; OXADIAZOLE DERIVATIVE; PURINERGIC RECEPTOR BLOCKING AGENT; QUINOLONE DERIVATIVE; STOP CODON;

ACUTE RESPIRATORY TRACT DISEASE; ANION TRANSPORT; ANTIBIOTIC THERAPY; ARTICLE; CHRONIC RESPIRATORY TRACT DISEASE; CLINICAL EFFECTIVENESS; DRUG RESPONSE; ENVIRONMENTAL FACTOR; EX VIVO STUDY; FAILURE TO THRIVE; FUNCTIONAL ASSESSMENT; GENE MUTATION; GENOTYPE; HUMAN; LUNG CYSTIC FIBROSIS; LUNG DISEASE; LUNG FIBROSIS; LUNG INFECTION; MONOGENIC DISORDER; NONHUMAN; PERSONALIZED MEDICINE; PHASE 3 CLINICAL TRIAL (TOPIC); PHASE 4 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN CONFORMATION; RARE DISEASE; BLOOD; CYSTIC FIBROSIS; DRUG EFFECTS; EVIDENCE BASED MEDICINE; FRAMESHIFT MUTATION; GENETICS; MORTALITY; PHENOTYPE; SEVERITY OF ILLNESS INDEX; STOP CODON; SWEAT GLAND;

AMINOGLYCOSIDES; AMINOPHENOLS; ANTI-BACTERIAL AGENTS; ANTI-INFLAMMATORY AGENTS; BIOLOGICAL MARKERS; CODON, NONSENSE; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; EVIDENCE-BASED MEDICINE; FRAMESHIFT MUTATION; GENISTEIN; HUMANS; INDIVIDUALIZED MEDICINE; OXADIAZOLES; PHENOTYPE; PURINERGIC ANTAGONISTS; QUINOLONES; RARE DISEASES; SEVERITY OF ILLNESS INDEX; SWEAT GLANDS;

EID: 84921487109     PISSN: 09546820     EISSN: 13652796     Source Type: Journal    
DOI: 10.1111/joim.12314     Document Type: Article

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