Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy-Reference-Cited by-同舟云学术

Assays for the Identification and Prioritization of Drug Candidates for Spinal Muscular Atrophy

Published:2014-08 Issue:6 Volume:12 Page:315-341
ISSN:1540-658X
Container-title:ASSAY and Drug Development Technologies
language:en
Short-container-title:ASSAY and Drug Development Technologies

Author:

Cherry Jonathan J.¹,Kobayashi Dione T.²,Lynes Maureen M.³⁴,Naryshkin Nikolai N.⁵,Tiziano Francesco Danilo⁶,Zaworski Phillip G.⁷,Rubin Lee L.³⁴,Jarecki Jill⁸

Affiliation:

1. Pfizer, Inc., Cambridge, Massachusetts.

2. Independent Consultant, San Francisco, California.

3. Department of Stem Cell and Regenerative Biology, Harvard University, Cambridge, Massachusetts.

4. Harvard Stem Cell Institute, Harvard University, Cambridge, Massachusetts.

5. PTC Therapeutics Inc., South Plainfield, New Jersey.

6. Istituto di Genetica Medica, Università Cattolica del Sacro Cuore, Roma, Italy.

7. PharmOptima LLC., Portage, Michigan.

8. Families of SMA, Elk Grove Village, Illinois.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/adt.2014.587

Reference227 articles.

1. From enigmatic to problematic: The new molecular genetics of childhood spinal muscular atrophy

2. Identification of Proximal Spinal Muscular Atrophy Carriers and Patients by Analysis of SMNT and SMNC Gene Copy Number

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

4. Autosomal dominant spinal muscular atrophy

5. Consensus Statement for Standard of Care in Spinal Muscular Atrophy

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