Literature DB >> 33135495

Batten disease: an expert update on agents in preclinical and clinical trials.

Margaux C Masten1, Jonathan W Mink1, Erika F Augustine1.   

Abstract

Keywords:  Batten disease; Disease-modifying therapy; gene therapy; neurodegenerative diseases; neuronal ceroid lipofuscinosis; pediatric

Year:  2020        PMID： 33135495      PMCID： PMC7736171          DOI： 10.1080/13543784.2020.1837110

Source DB:  PubMed          Journal:  Expert Opin Investig Drugs        ISSN： 1354-3784            Impact factor:   6.206


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  19 in total

1.  Clinical trials in rare disease: challenges and opportunities.

Authors:  Erika F Augustine; Heather R Adams; Jonathan W Mink
Journal:  J Child Neurol       Date:  2013-09       Impact factor: 1.987

2.  Considerations for the treatment of infantile neuronal ceroid lipofuscinosis (infantile Batten disease).

Authors:  Mark S Sands
Journal:  J Child Neurol       Date:  2013-09       Impact factor: 1.987

3.  Gemfibrozil, food and drug administration-approved lipid-lowering drug, increases longevity in mouse model of late infantile neuronal ceroid lipofuscinosis.

Authors:  Arunava Ghosh; Suresh Babu Rangasamy; Khushbu K Modi; Kalipada Pahan
Journal:  J Neurochem       Date:  2017-04-03       Impact factor: 5.372

4.  Short-Term Administration of Mycophenolate Is Well-Tolerated in CLN3 Disease (Juvenile Neuronal Ceroid Lipofuscinosis).

Authors:  Erika F Augustine; Christopher A Beck; Heather R Adams; Sara Defendorf; Amy Vierhile; Derek Timm; Jill M Weimer; Jonathan W Mink; Frederick J Marshall
Journal:  JIMD Rep       Date:  2018-06-20

Review 5.  In vivo tissue-tropism of adeno-associated viral vectors.

Authors:  Arun Srivastava
Journal:  Curr Opin Virol       Date:  2016-09-03       Impact factor: 7.090

6.  Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Authors:  Jinkuk Kim; Chunguang Hu; Christelle Moufawad El Achkar; Lauren E Black; Julie Douville; Austin Larson; Mary K Pendergast; Sara F Goldkind; Eunjung A Lee; Ashley Kuniholm; Aubrie Soucy; Jai Vaze; Nandkishore R Belur; Kristina Fredriksen; Iva Stojkovska; Alla Tsytsykova; Myriam Armant; Renata L DiDonato; Jaejoon Choi; Laura Cornelissen; Luis M Pereira; Erika F Augustine; Casie A Genetti; Kira Dies; Brenda Barton; Lucinda Williams; Benjamin D Goodlett; Bobbie L Riley; Amy Pasternak; Emily R Berry; Kelly A Pflock; Stephen Chu; Chantal Reed; Kimberly Tyndall; Pankaj B Agrawal; Alan H Beggs; P Ellen Grant; David K Urion; Richard O Snyder; Susan E Waisbren; Annapurna Poduri; Peter J Park; Al Patterson; Alessandra Biffi; Joseph R Mazzulli; Olaf Bodamer; Charles B Berde; Timothy W Yu
Journal:  N Engl J Med       Date:  2019-10-09       Impact factor: 91.245

7.  Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.

Authors:  Jui-Yun Lu; Hemanth R Nelvagal; Lingling Wang; Shari G Birnbaum; Jonathan D Cooper; Sandra L Hofmann
Journal:  Mol Genet Metab       Date:  2015-05-12       Impact factor: 4.797

8.  Longitudinal In Vivo Monitoring of the CNS Demonstrates the Efficacy of Gene Therapy in a Sheep Model of CLN5 Batten Disease.

Authors:  Nadia L Mitchell; Katharina N Russell; Martin P Wellby; Hollie E Wicky; Lucia Schoderboeck; Graham K Barrell; Tracy R Melzer; Steven J Gray; Stephanie M Hughes; David N Palmer
Journal:  Mol Ther       Date:  2018-07-17       Impact factor: 11.454

9.  Oral cysteamine bitartrate and N-acetylcysteine for patients with infantile neuronal ceroid lipofuscinosis: a pilot study.

Authors:  Sondra W Levin; Eva H Baker; Wadih M Zein; Zhongjian Zhang; Zenaide M N Quezado; Ning Miao; Andrea Gropman; Kurt J Griffin; Simona Bianconi; Goutam Chandra; Omar I Khan; Rafael C Caruso; Aiyi Liu; Anil B Mukherjee
Journal:  Lancet Neurol       Date:  2014-07-02       Impact factor: 44.182

10.  Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.

Authors:  M L Katz; G C Johnson; S B Leach; B G Williamson; J R Coates; R E H Whiting; D P Vansteenkiste; M S Whitney
Journal:  Gene Ther       Date:  2017-02-02       Impact factor: 5.250
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  1 in total

Review 1.  Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies.

Authors:  Dunhui Li; Craig Stewart McIntosh; Frank Louis Mastaglia; Steve Donald Wilton; May Thandar Aung-Htut
Journal:  Transl Neurodegener       Date:  2021-05-20       Impact factor: 8.014
  1 in total

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