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Discovery of a Novel Class of Survival Motor Neuron 2 Splicing Modifiers for the Treatment of Spinal Muscular Atrophy.
Pinard, Emmanuel; Green, Luke; Reutlinger, Michael; Weetall, Marla; Naryshkin, Nikolai A; Baird, John; Chen, Karen S; Paushkin, Sergey V; Metzger, Friedrich; Ratni, Hasane.
Afiliação
  • Pinard E; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development, Roche Innovation Center Basel , Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Green L; 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.
  • Naryshkin NA; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Baird J; PTC Therapeutics, Inc. , 100 Corporate Court, South Plainfield, New Jersey 07080, United States.
  • Chen KS; SMA Foundation , 888 Seventh Avenue, Suite 400, New York, New York 10019, United States.
  • Paushkin SV; SMA Foundation , 888 Seventh Avenue, Suite 400, New York, New York 10019, United States.
  • Metzger F; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development, Roche Innovation Center Basel , Grenzacherstrasse 124, 4070 Basel, Switzerland.
  • Ratni H; F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development, Roche Innovation Center Basel , Grenzacherstrasse 124, 4070 Basel, Switzerland.
J Med Chem ; 60(10): 4444-4457, 2017 05 25.
Article em En | MEDLINE | ID: mdl-28441483

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Benzamidas / RNA Mensageiro / Atrofia Muscular Espinal / Splicing de RNA / Proteína 2 de Sobrevivência do Neurônio Motor Idioma: En Ano de publicação: 2017 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Benzamidas / RNA Mensageiro / Atrofia Muscular Espinal / Splicing de RNA / Proteína 2 de Sobrevivência do Neurônio Motor Idioma: En Ano de publicação: 2017 Tipo de documento: Article