The discovery of medicines for rare diseases

Future Medicinal Chemistry

Volumn 6, Issue 9, 2014, Pages 987-1002

  Swinney, David C ^a   Xia, Shuangluo ^a  

^a Institute for Rare and Neglected Diseases Drug Discovery   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AGALSIDASE BETA; BEDAQUILINE; BOSUTINIB; CANAKINUMAB; CARGLUMIC ACID; CRIZOTINIB; DASATINIB; ECALLANTIDE; ECULIZUMAB; GALSULFASE; HOMOHARRINGTONINE; ICATIBANT; IDURONATE 2 SULFATASE; IMATINIB; IVACAFTOR; LARONIDASE; MIGLUSTAT; NELARABINE; NILOTINIB; NITISINONE; PONATINIB; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RILONACEPT; RUXOLITINIB; SAPROPTERIN; TALIGLUCERASE ALFA; UNINDEXED DRUG; VELAGLUCERASE ALFA; VEMURAFENIB; VORINOSTAT;

ASSAY; DRUG DEVELOPMENT; DRUG MECHANISM; DRUG TARGETING; FOOD AND DRUG ADMINISTRATION; GENETICS; HUMAN; INBORN ERROR OF METABOLISM; NEOPLASM; NONHUMAN; PHENOTYPE; PHYSIOLOGICAL PROCESS; PRIORITY JOURNAL; RARE DISEASE; REVIEW; TRANSLATIONAL RESEARCH; UNITED STATES; METABOLISM; RARE DISEASES;

DRUG DISCOVERY; HUMANS; PHENOTYPE; RARE DISEASES;

EID: 84905157309     PISSN: 17568919     EISSN: 17568927     Source Type: Journal    
DOI: 10.4155/fmc.14.65     Document Type: Review

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