Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)-Reference-Cited by-同舟云学术

Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Published:2018-07-25 Issue:15 Volume:61 Page:6501-6517
ISSN:0022-2623
Container-title:Journal of Medicinal Chemistry
language:en
Short-container-title:J. Med. Chem.

Author:

Ratni Hasane¹^ORCID,Ebeling Martin¹,Baird John²,Bendels Stefanie¹,Bylund Johan¹,Chen Karen S.³,Denk Nora¹,Feng Zhihua⁴,Green Luke¹,Guerard Melanie¹,Jablonski Philippe¹,Jacobsen Bjoern¹,Khwaja Omar¹,Kletzl Heidemarie¹,Ko Chien-Ping⁴,Kustermann Stefan¹,Marquet Anne¹,Metzger Friedrich¹,Mueller Barbara¹,Naryshkin Nikolai A.²,Paushkin Sergey V.³,Pinard Emmanuel¹,Poirier Agnès¹,Reutlinger Michael¹,Weetall Marla²,Zeller Andreas¹,Zhao Xin²,Mueller Lutz¹

Affiliation:

1. F. Hoffmann-La Roche Ltd., pRED, Pharma Research & Early Development, Roche Innovation Center Basel, Grenzacherstrasse 124, 4070 Basel, Switzerland

2. PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, New Jersey 07080, United States

3. SMA Foundation, 888 Seventh Avenue, Suite 400, New York, New York 10019, United States

4. Section of Neurobiology, Department of Biological Sciences, University of Southern California, Los Angeles, California 90089, United States

Funder

Spinal Muscular Atrophy Foundation

PTC Therapeutics

F. Hoffmann-La Roche AG

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.8b00741

Reference36 articles.

1. CLASSIFICATION OF SPINAL MUSCULAR ATROPHIES

2. The Neurobiology of Childhood Spinal Muscular Atrophy

3. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72 400 specimens

4. Spinal muscular atrophy: The role of SMN in axonal mRNA regulation

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