SHOULD patients with asymptomatic pompe disease be treated? A nationwide study in france-Reference-Cited by-同舟云学术

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SHOULD patients with asymptomatic pompe disease be treated? A nationwide study in france

Published:2015-04-02 Issue:6 Volume:51 Page:884-889
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle Nerve

Author:

Echaniz-Laguna Andoni¹,Carlier Robert-Yves²,Laloui Kenza³,Carlier Pierre³,Salort-Campana Emmanuelle⁴,Pouget Jean⁴,Laforet Pascal³

Affiliation:

1. Centre de Référence des Maladies Neuromusculaires, Département de Neurologie; Hôpital de Hautepierre 1; Avenue Molière 67098 Strasbourg France

2. Assistance Publique-Hôpitaux de Paris (APHP), Service d'imagerie médicale, CIC-IT handicap; Hôpital Poincaré; Garches France

3. Centre de Référence Neuromusculaire Paris-Est, Hôpital Pitié-Salpêtrière, and U974; Université Pierre et Marie Curie; Paris France

4. Centre de Référence des Maladies Neuromusculaires; Aix-Marseille Université, APHP; Marseille France

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Neurology (clinical),Physiology

Reference20 articles.

1. Pompe's disease;Ploeg;Lancet,2008

2. Recombinant human acid alfa-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007

3. A randomized study of alglucosidase alfa in late-onset Pompe's disease;Ploeg;N Engl J Med,2010

4. Open-label extension study following the Late-Onset Treatment Study (LOTS) of alglucosidase alfa;Ploeg;Mol Genet Metab,2012

5. Making diagnosis of Pompe disease at a presymptomatic stage: to treat or not to treat?;Laloui;Neurology,2011

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1. Enfermedad de Pompe del adulto: análisis de 13 pacientes;Medicina Clínica;2024-08

2. The Importance of Early Treatment of Inherited Neuromuscular Conditions;Journal of Neuromuscular Diseases;2024-03-05

3. HyperCKemia;Reference Module in Neuroscience and Biobehavioral Psychology;2024

4. Treatment Dilemma in Children with Late-Onset Pompe Disease;Genes;2023-01-30

5. Orphan drugs in treatment of rare neuromuscular disorders;Neurologie pro praxi;2021-04-30

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