Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

Human Molecular Genetics

Volumn 25, Issue 10, 2016, Pages 1885-1899

  Zhao, Xin ^a   Feng, Zhihua ^b   Ling, Karen K Y ^b   Mollin, Anna ^a   Sheedy, Josephine ^a   Yeh, Shirley ^a   Petruska, Janet ^a   Narasimhan, Jana ^a   Dakka, Amal ^a   Welch, Ellen M ^a   Karp, Gary ^a   Chen, Karen S ^e   Metzger, Friedrich ^c   Ratni, Hasane ^c   Lotti, Francesco ^d   Tisdale, Sarah ^d   Naryshkin, Nikolai A ^a   Pellizzoni, Livio ^d   Paushkin, Sergey ^e   Ko, Chien Ping ^b   Weetall, Marla ^a   more..

^a PTC THERAPEUTICS   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^c F HOFFMANN LA ROCHE LTD   (Switzerland)

^d NewYork Presbyterian Hospital   (United States)

^e SMA Foundation   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

SMALL NUCLEAR RIBONUCLEOPROTEIN; SMALL NUCLEAR RNA; SURVIVAL MOTOR NEURON PROTEIN 1; SURVIVAL MOTOR NEURON PROTEIN 2; ISOCOUMARIN DERIVATIVE; MOLECULAR LIBRARY; PIPERAZINE DERIVATIVE; SMN-C1 COMPOUND; SMN2 PROTEIN, MOUSE;

ADULT; ALTERNATIVE RNA SPLICING; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BLOOD; BODY WEIGHT; CENTRAL NERVOUS SYSTEM; CLINICAL EVALUATION; CLINICAL TRIAL; CONTROLLED STUDY; DRUG EFFICACY; EXON; GENE EXPRESSION; HUMAN; HUMAN CELL; LOCOMOTION; LONG TERM SURVIVAL; MOTONEURON; MOUSE; NEWBORN; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PHENOTYPE; PRIORITY JOURNAL; PROTEIN FUNCTION; PROTEIN SPLICING; RNA PROCESSING; SKIN; SPINAL CORD; SPINAL MUSCULAR ATROPHY; SURVIVAL; ADMINISTRATION AND DOSAGE; ANIMAL; DISEASE MODEL; DOSE RESPONSE; DRUG EFFECTS; GENETICS; METABOLISM; MOLECULAR LIBRARY; MONONUCLEAR CELL; MUSCULAR ATROPHY, SPINAL; PATHOLOGY; PHARMACOKINETICS; RNA SPLICING; TRANSGENIC MOUSE;

ALTERNATIVE SPLICING; ANIMALS; CENTRAL NERVOUS SYSTEM; DISEASE MODELS, ANIMAL; DOSE-RESPONSE RELATIONSHIP, DRUG; EXONS; HUMANS; ISOCOUMARINS; LEUKOCYTES, MONONUCLEAR; MICE; MICE, TRANSGENIC; MUSCULAR ATROPHY, SPINAL; PIPERAZINES; RNA SPLICING; SKIN; SMALL MOLECULE LIBRARIES; SURVIVAL OF MOTOR NEURON 2 PROTEIN;

EID: 84992198044     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddw062     Document Type: Article

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