Characteristics of Patients With Late-Onset Pompe Disease in France-Reference-Cited by-同舟云学术

Characteristics of Patients With Late-Onset Pompe Disease in France

Published:2023-08-29 Issue:9 Volume:101 Page:
ISSN:0028-3878
Container-title:Neurology
language:en
Short-container-title:Neurology

Author:

Lefeuvre Claire¹^ORCID,De Antonio Marie¹,Bouhour Francoise¹,Tard Celine¹,Salort-Campana Emmanuelle¹^ORCID,Lagrange Emmeline¹^ORCID,Behin Anthony¹,Sole Guilhem¹^ORCID,Noury Jean-Baptiste¹,Sacconi Sabrina¹,Magot Armelle¹,Nadaj-Pakleza Aleksandra¹,Lacour Arnaud¹,Beltran Stephane¹,Spinazzi Marco¹,Cintas Pascal¹,Renard Dimitri¹,Michaud Maud¹,Bedat-Millet Anne-Laure¹,Prigent Helene¹,Taouagh Nadjib¹,Arrassi Azzeddine¹,Hamroun Dalil¹^ORCID,Attarian Shahram¹^ORCID,Laforêt Pascal¹,Bouibede Fatima,Caillaud Catherine,Davion Jean-Baptiste,Deibener-Kaminsky Joelle,Demurger Florence,Durieu Isabelle,Echaniz-Laguna Andonin,Esselin Florence,Fournier Maxime,Froissart Roseline,Garcia Pierre Yves,Germain D.P.,Ghorab Karima,Nicolas Guillaume,Jaussaud Rolland,Krim Elsa,Magy Laurent,Masingue Marion,Minot-Myhié Marie-Christine,Nollet Sylvain,Not Adeline,Orlikowski David,Perez Thierry,Petiot Philippe,Praline Julien,Tai the Frédéric,Toquet Ségolène,Zagnoli Fabien, ,

Affiliation:

1. From the Neurology Department (C.L., N.T., P.L.), Raymond Poincaré University Hospital, Garches, APHP; Nord-Est-Ile-de-France Neuromuscular Reference Center (C.L., C.T., A.B., A.N.-P., M.M., H.P., N.T., A.A., P.L.), FHU PHENIX; Biostatistics Unit (DRCI) (M.D.A.), Clermont-Ferrand University Hospital; Service d'Electroneuromyographie et Pathologies Neuromusculaires (F.B.), Hospices Civils de Lyon; Inserm (C.T.), Lille University Hospital Center, U1172, Lille Neuroscience & Cognition, University...

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Neurology (clinical)

Link

https://www.neurology.org/doi/pdfdirect/10.1212/WNL.0000000000207547

Reference21 articles.

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2. Juvenile and adult-onset acid maltase deficiency in France: Genotype-phenotype correlation

3. Pompe's disease

4. Sibling phenotype concordance in classical infantile Pompe disease

5. Immunomodulatory gene therapy in lysosomal storage disorders;Koeberl DD;Curr Gene Ther,2009

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1. Characteristics of Patients With Late-Onset Pompe Disease in France;Neurology;2024-08-13

2. Bulbar muscle impairment in patients with late onset Pompe disease: Insight from the French Pompe registry;European Journal of Neurology;2024-08-07

3. Real‐life effectiveness 1 year after switching to avalglucosidase alfa in late‐onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study;European Journal of Neurology;2024-04-08

4. Characterization of neuromuscular performances in adults with late-onset Pompe disease: A control case cross-sectional study;Neuromuscular Disorders;2023-12