Literature DB >> 21094929

Where do we stand in enzyme replacement therapy in Pompe's disease?

A T van der Ploeg.   

Abstract

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Year:  2010        PMID: 21094929     DOI: 10.1016/j.nmd.2010.09.011

Source DB:  PubMed          Journal:  Neuromuscul Disord        ISSN: 0960-8966            Impact factor:   4.296


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  8 in total

1.  Therapeutic advances in the management of Pompe disease and other metabolic myopathies.

Authors:  Corrado Angelini; Anna Chiara Nascimbeni; Claudio Semplicini
Journal:  Ther Adv Neurol Disord       Date:  2013-09       Impact factor: 6.570

Review 2.  Enzyme replacement therapy for Pompe disease.

Authors:  Corrado Angelini; Claudio Semplicini
Journal:  Curr Neurol Neurosci Rep       Date:  2012-02       Impact factor: 5.081

3.  Autophagy in Natural History and After ERT in Glycogenosis Type II.

Authors:  Corrado Angelini; Anna C Nascimbeni; Marina Fanin
Journal:  JIMD Rep       Date:  2015-02-25

4.  Observational clinical study in juvenile-adult glycogenosis type 2 patients undergoing enzyme replacement therapy for up to 4 years.

Authors:  C Angelini; C Semplicini; S Ravaglia; B Bembi; S Servidei; E Pegoraro; M Moggio; M Filosto; E Sette; G Crescimanno; P Tonin; R Parini; L Morandi; G Marrosu; G Greco; O Musumeci; G Di Iorio; G Siciliano; M A Donati; F Carubbi; M Ermani; T Mongini; A Toscano
Journal:  J Neurol       Date:  2011-11-12       Impact factor: 4.849

5.  Peak Jump Power Reflects the Degree of Ambulatory Ability in Patients with Mitochondrial and Other Rare Diseases.

Authors:  Christopher Newell; Barbara Ramage; Alberto Nettel-Aguirre; Ion Robu; Aneal Khan
Journal:  JIMD Rep       Date:  2016-09-13

Review 6.  Progress and problems in muscle glycogenoses.

Authors:  S DiMauro; R Spiegel
Journal:  Acta Myol       Date:  2011-10

7.  Bioimpedance Phase Angle as a Prognostic Tool in Late-Onset Pompe Disease: A Single-Centre Prospective Study With a 15-year Follow-Up.

Authors:  Sabrina Ravaglia; Rachele de Giuseppe; Annalisa Carlucci; Susan Jehne; Grazia Crescimanno; Lara Ahmad; Matteo Paoletti; Gabriele Clemente; Anna Pichiecchio; Rosella Bazzano; Serena Cirio; Enza Maria Valente; Cesare Danesino; Paola De Filippi; Alice Tartara; Hellas Cena
Journal:  Front Cell Dev Biol       Date:  2022-02-18

8.  Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.

Authors:  Merel Stok; Helen de Boer; Marshall W Huston; Edwin H Jacobs; Onno Roovers; Trudi P Visser; Holger Jahr; Dirk J Duncker; Elza D van Deel; Arnold J J Reuser; Niek P van Til; Gerard Wagemaker
Journal:  Mol Ther Methods Clin Dev       Date:  2020-05-04       Impact factor: 6.698

  8 in total

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