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Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA).
Ratni, Hasane; Ebeling, Martin; Baird, John; Bendels, Stefanie; Bylund, Johan; Chen, Karen S; Denk, Nora; Feng, Zhihua; Green, Luke; Guerard, Melanie; Jablonski, Philippe; Jacobsen, Bjoern; Khwaja, Omar; Kletzl, Heidemarie; Ko, Chien-Ping; Kustermann, Stefan; Marquet, Anne; Metzger, Friedrich; Mueller, Barbara; Naryshkin, Nikolai A; Paushkin, Sergey V; Pinard, Emmanuel; Poirier, Agnès; Reutlinger, Michael; Weetall, Marla; Zeller, Andreas; Zhao, Xin; Mueller, Lutz.
Affiliation
  • Ratni H; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Ebeling M; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Baird J; PTC Therapeutics, Inc. , 100 Corporate Court , South Plainfield , New Jersey 07080 , United States.
  • Bendels S; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Bylund J; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Chen KS; SMA Foundation , 888 Seventh Avenue, Suite 400 , New York , New York 10019 , United States.
  • Denk N; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Feng Z; Section of Neurobiology, Department of Biological Sciences , University of Southern California , Los Angeles , California 90089 , United States.
  • Green L; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Guerard M; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Jablonski P; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Jacobsen B; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Khwaja O; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Kletzl H; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Ko CP; Section of Neurobiology, Department of Biological Sciences , University of Southern California , Los Angeles , California 90089 , United States.
  • Kustermann S; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Marquet A; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Metzger F; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Mueller B; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • 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.
  • Pinard E; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Poirier A; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Reutlinger M; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Weetall M; PTC Therapeutics, Inc. , 100 Corporate Court , South Plainfield , New Jersey 07080 , United States.
  • Zeller A; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
  • Zhao X; PTC Therapeutics, Inc. , 100 Corporate Court , South Plainfield , New Jersey 07080 , United States.
  • Mueller L; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development , Roche Innovation Center Basel , Grenzacherstrasse 124 , 4070 Basel , Switzerland.
J Med Chem ; 61(15): 6501-6517, 2018 Aug 09.
Article in En | MEDLINE | ID: mdl-30044619
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pyrimidines / Azo Compounds / Muscular Atrophy, Spinal / RNA Splicing / Survival of Motor Neuron 2 Protein / Drug Discovery Limits: Animals / Humans Language: En Journal: J Med Chem Journal subject: QUIMICA Year: 2018 Document type: Article Affiliation country: Country of publication:
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pyrimidines / Azo Compounds / Muscular Atrophy, Spinal / RNA Splicing / Survival of Motor Neuron 2 Protein / Drug Discovery Limits: Animals / Humans Language: En Journal: J Med Chem Journal subject: QUIMICA Year: 2018 Document type: Article Affiliation country: Country of publication: