Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy-Reference-Cited by-同舟云学术

Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy

Published:2016-07-06 Issue:13 Volume:59 Page:6086-6100
ISSN:0022-2623
Container-title:Journal of Medicinal Chemistry
language:en
Short-container-title:J. Med. Chem.

Author:

Ratni Hasane¹,Karp Gary M.²,Weetall Marla²,Naryshkin Nikolai A.²,Paushkin Sergey V.³,Chen Karen S.³,McCarthy Kathleen D.³,Qi Hongyan²,Turpoff Anthony²,Woll Matthew G.²,Zhang Xiaoyan²,Zhang Nanjing²,Yang Tianle²,Dakka Amal²,Vazirani Priya²,Zhao Xin²,Pinard Emmanuel¹,Green Luke¹,David-Pierson Pascale¹,Tuerck Dietrich¹,Poirier Agnes¹,Muster Wolfgang¹,Kirchner Stephan¹,Mueller Lutz¹,Gerlach Irene¹,Metzger Friedrich¹

Affiliation:

1. pRED, Pharma Research & Early Development, F. Hoffmann-La Roche Ltd., 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

Funder

Spinal Muscular Atrophy Foundation

F. Hoffmann-La Roche

PTC Therapeutics Inc.

Publisher

American Chemical Society (ACS)

Subject

Drug Discovery,Molecular Medicine

Link

https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.6b00459

Reference22 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

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

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