Muscle Proteomic Profile before and after Enzyme Replacement Therapy in Late-Onset Pompe Disease

Mutations in the acidic alpha-glucosidase (GAA) coding gene cause Pompe disease. Late-onset Pompe disease (LOPD) is characterized by progressive proximal and axial muscle weakness and atrophy, causing respiratory failure. Enzyme replacement therapy (ERT), based on recombinant human GAA infusions, is...

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Détails bibliographiques
Publié dans:Int J Mol Sci
Auteurs principaux: Moriggi, Manuela, Capitanio, Daniele, Torretta, Enrica, Barbacini, Pietro, Bragato, Cinzia, Sartori, Patrizia, Moggio, Maurizio, Maggi, Lorenzo, Mora, Marina, Gelfi, Cecilia
Format: Artigo
Langue:Inglês
Publié: MDPI 2021
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC8001152/
https://ncbi.nlm.nih.gov/pubmed/33799647
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22062850
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