Literature DB >> 23887636

Letter to the Editors: Concerning "CRIM-negative Pompe disease patients with satisfactory clinical outcomes on enzyme replacement therapy" by Al Khallaf et al.

Sean N Prater1, Suhrad G Banugaria, Claire Morgan, Crystal C Sung, Amy S Rosenberg, Priya S Kishnani.   

Abstract

Entities:  

Year:  2013        PMID： 23887636      PMCID： PMC4353589          DOI： 10.1007/s10545-013-9637-8

Source DB:  PubMed          Journal:  J Inherit Metab Dis        ISSN： 0141-8955            Impact factor:   4.982


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  7 in total

1.  Atypical immunologic response in a patient with CRIM-negative Pompe disease.

Authors:  Mary-Alice Abbott; Sean N Prater; Suhrad G Banugaria; Susan M Richards; Sarah P Young; Amy S Rosenberg; Priya S Kishnani
Journal:  Mol Genet Metab       Date:  2011-08-11       Impact factor: 4.797

2.  CRIM-negative infantile Pompe disease: 42-month treatment outcome.

Authors:  Marianne Rohrbach; Andrea Klein; Alice Köhli-Wiesner; Dorothe Veraguth; Ianina Scheer; Christian Balmer; Roger Lauener; Matthias R Baumgartner
Journal:  J Inherit Metab Dis       Date:  2010-09-30       Impact factor: 4.982

3.  Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants.

Authors:  Priya S Kishnani; Paula C Goldenberg; Stephanie L DeArmey; James Heller; Danny Benjamin; Sarah Young; Deeksha Bali; Sue Ann Smith; Jennifer S Li; Hanna Mandel; Dwight Koeberl; Amy Rosenberg; Y-T Chen
Journal:  Mol Genet Metab       Date:  2010-01       Impact factor: 4.797

4.  The emerging phenotype of long-term survivors with infantile Pompe disease.

Authors:  Sean N Prater; Suhrad G Banugaria; Stephanie M DeArmey; Eleanor G Botha; Erin M Stege; Laura E Case; Harrison N Jones; Chanika Phornphutkul; Raymond Y Wang; Sarah P Young; Priya S Kishnani
Journal:  Genet Med       Date:  2012-04-26       Impact factor: 8.822

5.  The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.

Authors:  Suhrad G Banugaria; Sean N Prater; Yiu-Ki Ng; Joyce A Kobori; Richard S Finkel; Roger L Ladda; Yuan-Tsong Chen; Amy S Rosenberg; Priya S Kishnani
Journal:  Genet Med       Date:  2011-08       Impact factor: 8.822

6.  Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.

Authors:  Yoav H Messinger; Nancy J Mendelsohn; William Rhead; David Dimmock; Eli Hershkovitz; Michael Champion; Simon A Jones; Rebecca Olson; Amy White; Cara Wells; Deeksha Bali; Laura E Case; Sarah P Young; Amy S Rosenberg; Priya S Kishnani
Journal:  Genet Med       Date:  2012-01       Impact factor: 8.822

7.  Bortezomib in the rapid reduction of high sustained antibody titers in disorders treated with therapeutic protein: lessons learned from Pompe disease.

Authors:  Suhrad G Banugaria; Sean N Prater; Judeth K McGann; Jonathan D Feldman; Jesse A Tannenbaum; Carrie Bailey; Renuka Gera; Robert L Conway; David Viskochil; Joyce A Kobori; Amy S Rosenberg; Priya S Kishnani
Journal:  Genet Med       Date:  2012-10-11       Impact factor: 8.822
  7 in total
  5 in total

1.  Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.

Authors:  Zoheb B Kazi; Ankit K Desai; Kathryn L Berrier; R Bradley Troxler; Raymond Y Wang; Omar A Abdul-Rahman; Pranoot Tanpaiboon; Nancy J Mendelsohn; Eli Herskovitz; David Kronn; Michal Inbar-Feigenberg; Catherine Ward-Melver; Michelle Polan; Punita Gupta; Amy S Rosenberg; Priya S Kishnani
Journal:  JCI Insight       Date:  2017-08-17

2.  CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy.

Authors:  Kathryn L Berrier; Zoheb B Kazi; Sean N Prater; Deeksha S Bali; Jennifer Goldstein; Mihaela C Stefanescu; Catherine W Rehder; Eleanor G Botha; Carolyn Ellaway; Kaustuv Bhattacharya; Anna Tylki-Szymanska; Nesrin Karabul; Amy S Rosenberg; Priya S Kishnani
Journal:  Genet Med       Date:  2015-03-05       Impact factor: 8.822

3.  Letter to the Editors: Concerning "Divergent clinical outcomes of alphaglucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state" by Takashi M et al.

Authors:  Rita Ortolano; Federico Baronio; Riccardo Masetti; Arcangelo Prete; Alessandra Cassio; Andrea Pession
Journal:  Mol Genet Metab Rep       Date:  2017-03-07

4.  Characterization of immune response in Cross-Reactive Immunological Material (CRIM)-positive infantile Pompe disease patients treated with enzyme replacement therapy.

Authors:  Ankit K Desai; Zoheb B Kazi; Deeksha S Bali; Priya S Kishnani
Journal:  Mol Genet Metab Rep       Date:  2019-05-10

Review 5.  Immune responses to alglucosidase in infantile Pompe disease: recommendations from an Italian pediatric expert panel.

Authors:  Vincenza Gragnaniello; Federica Deodato; Serena Gasperini; Maria Alice Donati; Clementina Canessa; Simona Fecarotta; Antonia Pascarella; Giuseppe Spadaro; Daniela Concolino; Alberto Burlina; Giancarlo Parenti; Pietro Strisciuglio; Agata Fiumara; Roberto Della Casa
Journal:  Ital J Pediatr       Date:  2022-03-05       Impact factor: 2.638
  5 in total

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