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Literature DB >> 34338226

Assuring long-term safety of highly effective gene-modulating therapeutics for rare diseases.

Thomas O Crawford^1,2, Charlotte J Sumner^1,3.

Abstract

Entities: Chemical

Mesh：

Year: 2021 PMID： 34338226 PMCID： PMC8321560 DOI： 10.1172/JCI152817

Source DB: PubMed Journal: J Clin Invest ISSN： 0021-9738 Impact factor: 19.456

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20 in total

1. Postmarketing surveillance--lack of vigilance, lack of trust.

Authors: Phil B Fontanarosa; Drummond Rennie; Catherine D DeAngelis
Journal: JAMA Date: 2004-11-22 Impact factor: 56.272

2. Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series.

Authors: Deepa H Chand; Craig Zaidman; Kapil Arya; Rachel Millner; Michelle A Farrar; Fiona E Mackie; Natalie L Goedeker; Vikas R Dharnidharka; Raja Dandamudi; Sandra P Reyna
Journal: J Pediatr Date: 2020-11-28 Impact factor: 4.406

3. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial.

Authors: John W Day; Richard S Finkel; Claudia A Chiriboga; Anne M Connolly; Thomas O Crawford; Basil T Darras; Susan T Iannaccone; Nancy L Kuntz; Loren D M Peña; Perry B Shieh; Edward C Smith; Jennifer M Kwon; Craig M Zaidman; Meredith Schultz; Douglas E Feltner; Sitra Tauscher-Wisniewski; Haojun Ouyang; Deepa H Chand; Douglas M Sproule; Thomas A Macek; Jerry R Mendell
Journal: Lancet Neurol Date: 2021-03-17 Impact factor: 44.182

4. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors: Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal: N Engl J Med Date: 2017-11-02 Impact factor: 91.245

5. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Authors: Richard S Finkel; Eugenio Mercuri; Basil T Darras; Anne M Connolly; Nancy L Kuntz; Janbernd Kirschner; Claudia A Chiriboga; Kayoko Saito; Laurent Servais; Eduardo Tizzano; Haluk Topaloglu; Már Tulinius; Jacqueline Montes; Allan M Glanzman; Kathie Bishop; Z John Zhong; Sarah Gheuens; C Frank Bennett; Eugene Schneider; Wildon Farwell; Darryl C De Vivo
Journal: N Engl J Med Date: 2017-11-02 Impact factor: 91.245

6. Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA.

Authors: Lingling Kong; David O Valdivia; Christian M Simon; Cera W Hassinan; Nicolas Delestrée; Daniel M Ramos; Jae Hong Park; Celeste M Pilato; Xixi Xu; Melissa Crowder; Chloe C Grzyb; Zachary A King; Marco Petrillo; Kathryn J Swoboda; Crystal Davis; Cathleen M Lutz; Alexander H Stephan; Xin Zhao; Marla Weetall; Nikolai A Naryshkin; Thomas O Crawford; George Z Mentis; Charlotte J Sumner
Journal: Sci Transl Med Date: 2021-01-27 Impact factor: 17.956

7. Risdiplam in Type 1 Spinal Muscular Atrophy.

Authors: Giovanni Baranello; Basil T Darras; John W Day; Nicolas Deconinck; Andrea Klein; Riccardo Masson; Eugenio Mercuri; Kristy Rose; Muna El-Khairi; Marianne Gerber; Ksenija Gorni; Omar Khwaja; Heidemarie Kletzl; Renata S Scalco; Timothy Seabrook; Paulo Fontoura; Laurent Servais
Journal: N Engl J Med Date: 2021-02-24 Impact factor: 91.245

8. Regulation of SMN protein stability.

Authors: Barrington G Burnett; Eric Muñoz; Animesh Tandon; Deborah Y Kwon; Charlotte J Sumner; Kenneth H Fischbeck
Journal: Mol Cell Biol Date: 2008-12-22 Impact factor: 4.272

9. Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect.

Authors: Tatiana O Gavrilina; Vicki L McGovern; Eileen Workman; Thomas O Crawford; Rocky G Gogliotti; Christine J DiDonato; Umrao R Monani; Glenn E Morris; Arthur H M Burghes
Journal: Hum Mol Genet Date: 2008-01-04 Impact factor: 6.150