Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy

Journal of Medicinal Chemistry

Volumn 60, Issue 10, 2017, Pages 4444-4457

  Pinard, Emmanuel ^a   Green, Luke ^a   Reutlinger, Michael ^a   Weetall, Marla ^b   Naryshkin, Nikolai A ^b   Baird, John ^b   Chen, Karen S ^c   Paushkin, Sergey V ^c   Metzger, Friedrich ^a   Ratni, Hasane ^a  

^a F HOFFMANN LA ROCHE LTD   (Switzerland)

^b PTC THERAPEUTICS   (United States)

^c SMA Foundation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2 FLUORO N (2 METHYLIMIDAZO[1,2 A]PYRAZIN 6 YL) 4 PIPERAZIN 1 YL BENZAMIDE; 2 FLUORO N (2 METHYLIMIDAZO[1,2 A]PYRAZIN 6 YL) 4 [3 METHYLPIPERAZIN 1 YL]BENZAMIDE; 4 [3 ETHYLPIPERAZIN 1 YL] 2 FLUORO N (2 METHYLIMIDAZO[1,2 A]PYRAZIN 6 YL)BENZAMIDE; 4 [3 ETHYLPIPERAZIN 1 YL] 2 FLUORO N (2 METHYLIMIDAZO[1,2A]PYRAZIN 6 YL)BENZAMIDE; BENZAMIDE DERIVATIVE; N (6,8 DIMETHYLIMIDAZO[1,2 A]PYRAZIN 2 YL) 2 FLUORO 4 (3 METHYLPIPERAZIN 1 YL)BENZAMIDE; N (6,8 DIMETHYLIMIDAZO[1,2 A]PYRAZIN 2 YL) 2 FLUORO 4 (4 METHYLPIPERAZIN 1 YL)BENZAMIDE; N (6,8 DIMETHYLIMIDAZO[1,2 A]PYRAZIN 2 YL) 4 (4 METHYLPIPERAZIN 1 YL)BENZAMIDE; N (6,8 DIMETHYLIMIDAZO[1,2 A]PYRAZIN 2 YL) 4 PIPERAZIN 1 YL BENZAMIDE; PROTEIN INHIBITOR; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG; MESSENGER RNA;

ADULT; ALTERNATIVE RNA SPLICING; ANIMAL EXPERIMENT; ANIMAL MODEL; AREA UNDER THE CURVE; ARTICLE; CONTROLLED STUDY; DRUG DESIGN; DRUG POTENCY; DRUG SAFETY; DRUG SCREENING; DRUG SYNTHESIS; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; IN VITRO STUDY; IN VIVO STUDY; LIPOPHILICITY; METABOLIC STABILITY; MOUSE; NONHUMAN; NUCLEOPHILICITY; PHYSICAL CHEMISTRY; PROTEIN CONFORMATION; PROTON NUCLEAR MAGNETIC RESONANCE; SPINAL MUSCULAR ATROPHY; TREATMENT DURATION; ANIMAL; CHEMISTRY; DRUG EFFECTS; GENETICS; MOLECULAR MODEL; MUSCULAR ATROPHY, SPINAL; RNA SPLICING;

ANIMALS; BENZAMIDES; DRUG DESIGN; MICE; MODELS, MOLECULAR; MUSCULAR ATROPHY, SPINAL; RNA SPLICING; RNA, MESSENGER; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 85019659898     PISSN: 00222623     EISSN: 15204804     Source Type: Journal    
DOI: 10.1021/acs.jmedchem.7b00406     Document Type: Article

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