Drug discovery and development for Huntington's disease - An orphan indication with high medical need

IDrugs

Volumn 11, Issue 9, 2008, Pages 653-660

  Heitz, F ^a   La Rosa, S ^a   Gonzalez Couto, E ^a   Gaviraghi, G ^a   Terstappen, G C ^a  

^a Siena Biotech SpA   (Italy)

Author keywords

Aggregation; HD; Huntington's disease; Orphan disease; Phenotypic assay; Transcriptional dysregulation

Indexed keywords

2 AMINOTHIAZOLE DERIVATIVE; 4 (1 AMINOETHYL) N (4 PYRIDYL)CYCLOHEXANECARBOXAMIDE; A 28; ACR 16; BPT 2; CONGO RED; CREATINE; DIMEBON; EPIGALLOCATECHIN GALLATE; ICOSAPENTAENOIC ACID ETHYL ESTER; INDOLEPROPIONIC ACID; ISTRADEFYLLINE; KANEKAQ10; LAMOTRIGINE; MEMANTINE; MERCAPTAMINE; MINOCYCLINE; OLESOXIME; ORPHAN DRUG; OXIGON; PBT 2; PGL 135; RAPAMYCIN; REMACEMIDE; RESVERATROL; RILUZOLE; SMER 10; TETRABENAZINE; TREHALOSE; TRO 19622; TRO 47498; UBIDECARENONE; UNCLASSIFIED DRUG;

ALZHEIMER DISEASE; CLINICAL TRIAL; DEGENERATIVE DISEASE; DRUG EFFICACY; DRUG MECHANISM; DRUG TOLERABILITY; HUMAN; HUNTINGTON CHOREA; MENTAL CAPACITY; MOTOR CONTROL; NONHUMAN; PALLIATIVE THERAPY; RARE DISEASE; REVIEW;

ANIMALS; CLINICAL TRIALS AS TOPIC; DRUG DESIGN; DRUG EVALUATION, PRECLINICAL; DRUGS, INVESTIGATIONAL; HUMANS; HUNTINGTON DISEASE; NEUROPROTECTIVE AGENTS; ORPHAN DRUG PRODUCTION; RARE DISEASES;

EID: 51049090179     PISSN: 13697056     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (5)

1. Gonzalez-Couto E, Matteoni S, Gotta S, Magnoni L, Heitz F, Raggiaschi R, Terstappen GC, Kremer A: Huntington's disease: From experimental results to interaction networks, pathopathway construction and disease hypothesis. BMC Syst Biol (2007) 1(Suppl 1):P45.
2. Harjes P, Wanker EE: The hunt for huntingtin function: Interaction partners tell many different stories. Trends Biochem Sci (2003) 28(8):425-433.
3. Mangiarini L, Sathasivam K, Seller M, Cozens B, Harper A, Hetherington C, Lawton M, Trottier Y, Lehrach H, Davies SW, Bates GP: Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice. Cell (1996) 87(3):493-506.
4. Ryu H, Ferrante RJ: Emerging chemotherapeutic strategies for Huntington's disease. Expert Opin Emerg Drugs (2005) 10(2):345-363.
5. Terstappen GC, Schluepen C, Raggiaschi R, Gaviraghi G: Target deconvolution strategies in drug discovery. Nat Rev Drug Discov (2007) 6(11):891-903.