دورية أكاديمية
Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024.
| العنوان: | Start, switch and stop (triple-S) criteria for enzyme replacement therapy of late-onset Pompe disease: European Pompe Consortium recommendation update 2024. |
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| المؤلفون: | Schoser, Benedikt, van der Beek, Nadine A M E, Broomfield, Alexander, Brusse, Esther, Diaz-Manera, Jordi, Hahn, Andreas, Hundsberger, Thomas, Kornblum, Cornelia, Kruijshaar, Michelle, Laforet, Pascal, Mengel, Eugen, Mongini, Tiziana, Orlikowski, David, Parenti, Giancarlo, Pijnappel, W W M Pim, Roberts, Mark, Scherer, Thomas, Toscano, Antonio, Vissing, John, van den Hout, Johanna M P, van Doorn, Pieter A, Wenninger, Stephan, van der Ploeg, Ans T |
| المصدر: | Eur J Neurol ; ISSN:1468-1331 |
| بيانات النشر: | Wiley |
| سنة النشر: | 2024 |
| المجموعة: | PubMed Central (PMC) |
| مصطلحات موضوعية: | EPOC, Pompe disease, enzyme replacement therapy, late‐onset, therapy switch |
| الوصف: | Two novel enzyme replacement therapies (ERTs), studied in phase 3 trials in late-onset Pompe patients, reached marketing authorization by the European Medicines Agency in 2022 and 2023. The European Pompe Consortium (EPOC) updates and extends the scope of the 2017 recommendations for starting, switching and stopping ERT. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | https://doi.org/10.1111/ene.16383Test; https://pubmed.ncbi.nlm.nih.gov/38873957Test |
| DOI: | 10.1111/ene.16383 |
| الإتاحة: | https://doi.org/10.1111/ene.16383Test https://pubmed.ncbi.nlm.nih.gov/38873957Test |
| حقوق: | © 2024 The Author(s). European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology. |
| رقم الانضمام: | edsbas.5C758F7A |
| قاعدة البيانات: | BASE |
| DOI: | 10.1111/ene.16383 |
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