Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA)-Reference-Cited by-同舟云学术

Discovery of Small Molecule Splicing Modulators of Survival Motor Neuron-2 (SMN2) for the Treatment of Spinal Muscular Atrophy (SMA)

Published:2018-11-08 Issue:24 Volume:61 Page:11021-11036
ISSN:0022-2623
Container-title:Journal of Medicinal Chemistry
language:en
Short-container-title:J. Med. Chem.

Author:

Cheung Atwood K.¹^ORCID,Hurley Brian¹,Kerrigan Ryan¹,Shu Lei¹,Chin Donovan N.¹,Shen Yiping¹,O’Brien Gary¹,Sung Moo Je¹,Hou Ying¹,Axford Jake¹,Cody Emma¹,Sun Robert¹,Fazal Aleem¹,Fridrich Cary¹,Sanchez Carina C.¹,Tomlinson Ronald C.¹,Jain Monish¹,Deng Lin¹,Hoffmaster Keith¹,Song Cheng¹,Van Hoosear Mailin¹,Shin Youngah¹,Servais Rebecca¹,Towler Christopher²,Hild Marc¹,Curtis Daniel¹,Dietrich William F.¹,Hamann Lawrence G.¹,Briner Karin¹,Chen Karen S.³,Kobayashi Dione³,Sivasankaran Rajeev¹,Dales Natalie A.¹

Affiliation:

1. Novartis Institutes for BioMedical Research, 250 Massachusetts Avenue, Cambridge, Massachusetts 02139, United States

2. Novartis Pharmaceuticals, 250 Massachusetts Avenue, Cambridge, Massachusetts 02139, United States

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

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Molecular Medicine

Link

http://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.8b01291

Reference39 articles.

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

2. Spinal muscular atrophy

3. Targeting RNA-splicing for SMA treatment

4. Spinal Muscular Atrophy

5. Identification and characterization of a spinal muscular atrophy-determining gene

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