Literature DB >> 31597016

Drug Regulation in the Era of Individualized Therapies.

Janet Woodcock1, Peter Marks1.   

Abstract

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Year:  2019        PMID： 31597016     DOI： 10.1056/NEJMe1911295

Source DB:  PubMed          Journal:  N Engl J Med        ISSN： 0028-4793            Impact factor:   91.245


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  11 in total

Review 1.  Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.

Authors:  Jill A Morris; Chris H Boshoff; Nina F Schor; Ling M Wong; Guangping Gao; Beverly L Davidson
Journal:  Mol Ther       Date:  2021-09-20       Impact factor: 11.454

Review 2.  Precision medicine for genetic epilepsy on the horizon: Recent advances, present challenges, and suggestions for continued progress.

Authors:  Juliet K Knowles; Ingo Helbig; Cameron S Metcalf; Laura S Lubbers; Lori L Isom; Scott Demarest; Ethan M Goldberg; Alfred L George; Holger Lerche; Sarah Weckhuysen; Vicky Whittemore; Samuel F Berkovic; Daniel H Lowenstein
Journal:  Epilepsia       Date:  2022-07-17       Impact factor: 6.740

Review 3.  Mechanisms of disease-associated SINE-VNTR-Alus.

Authors:  Abigail L Pfaff; Lewis M Singleton; Sulev Kõks
Journal:  Exp Biol Med (Maywood)       Date:  2022-04-06

4.  Long-Term Patient-Customized Therapy for a Pathogenic EPO Mutation.

Authors:  Ayesha Ejaz; Alper Ozcan; Ekrem Unal; Musa Karakukcu; Vijay G Sankaran
Journal:  Med (N Y)       Date:  2020-11-09

Review 5.  Randomized Controlled Trials Versus Real World Evidence: Neither Magic Nor Myth.

Authors:  Hans-Georg Eichler; Francesco Pignatti; Brigitte Schwarzer-Daum; Ana Hidalgo-Simon; Irmgard Eichler; Peter Arlett; Anthony Humphreys; Spiros Vamvakas; Nikolai Brun; Guido Rasi
Journal:  Clin Pharmacol Ther       Date:  2020-11-12       Impact factor: 6.875

Review 6.  Precision Reimbursement for Precision Medicine: Using Real-World Evidence to Evolve From Trial-and-Project to Track-and-Pay to Learn-and-Predict.

Authors:  Hans-Georg Eichler; Mark Trusheim; Brigitte Schwarzer-Daum; Kay Larholt; Markus Zeitlinger; Martin Brunninger; Michael Sherman; David Strutton; Gigi Hirsch
Journal:  Clin Pharmacol Ther       Date:  2021-11-17       Impact factor: 6.903

Review 7.  Ethical Issues in Care and Treatment of Neuronal Ceroid Lipofuscinoses (NCL)-A Personal View.

Authors:  Alfried Kohlschütter
Journal:  Front Neurol       Date:  2021-06-25       Impact factor: 4.003

8.  Genomic research delivering on promises: From rejuvenation to vaccines and pharmacogenetics.

Authors:  Alain Li Wan Po
Journal:  J Clin Pharm Ther       Date:  2020-03-18       Impact factor: 2.512

Review 9.  Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era.

Authors:  Dunhui Li; Frank L Mastaglia; Sue Fletcher; Steve D Wilton
Journal:  Med Res Rev       Date:  2020-08-06       Impact factor: 12.944

10.  Toward a new framework for the development of individualized therapies.

Authors:  Peter Marks; Celia Witten
Journal:  Gene Ther       Date:  2020-04-02       Impact factor: 5.250
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