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Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy.
Ratni, Hasane; Karp, Gary M; Weetall, Marla; Naryshkin, Nikolai A; Paushkin, Sergey V; Chen, Karen S; McCarthy, Kathleen D; Qi, Hongyan; Turpoff, Anthony; Woll, Matthew G; Zhang, Xiaoyan; Zhang, Nanjing; Yang, Tianle; Dakka, Amal; Vazirani, Priya; Zhao, Xin; Pinard, Emmanuel; Green, Luke; David-Pierson, Pascale; Tuerck, Dietrich; Poirier, Agnes; Muster, Wolfgang; Kirchner, Stephan; Mueller, Lutz; Gerlach, Irene; Metzger, Friedrich.
Afiliación
  • Ratni H; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Karp GM; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Weetall M; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Naryshkin NA; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Paushkin SV; SMA Foundation , 888 Seventh Avenue, Suite 400, New York, New York 10019, United States.
  • Chen KS; SMA Foundation , 888 Seventh Avenue, Suite 400, New York, New York 10019, United States.
  • McCarthy KD; SMA Foundation , 888 Seventh Avenue, Suite 400, New York, New York 10019, United States.
  • Qi H; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Turpoff A; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Woll MG; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Zhang X; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Zhang N; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Yang T; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Dakka A; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Vazirani P; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Zhao X; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Pinard E; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Green L; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • David-Pierson P; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Tuerck D; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Poirier A; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Muster W; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Kirchner S; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Mueller L; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Gerlach I; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Metzger F; pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd. , Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland.
J Med Chem ; 59(13): 6086-100, 2016 07 14.
Article en En | MEDLINE | ID: mdl-27299419
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Pirimidinonas / ARN Mensajero / Atrofia Muscular Espinal / Empalme Alternativo / Proteína 2 para la Supervivencia de la Neurona Motora Idioma: En Revista: J Med Chem Asunto de la revista: QUIMICA Año: 2016 Tipo del documento: Article
Texto completo
Imprimir
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Pirimidinonas / ARN Mensajero / Atrofia Muscular Espinal / Empalme Alternativo / Proteína 2 para la Supervivencia de la Neurona Motora Idioma: En Revista: J Med Chem Asunto de la revista: QUIMICA Año: 2016 Tipo del documento: Article