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Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy
Pompe disease is a neuromuscular disease caused by a deficiency of the lysosomal enzyme acid alpha-glucosidase leading to lysosomal and cytoplasmic glycogen accumulation in neurons and striated muscle. In the decade since availability of first-generation enzyme replacement therapy (ERT) a better und...
में बचाया:
| में प्रकाशित: | Ann Transl Med |
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| मुख्य लेखकों: | , , , , , , , , , |
| स्वरूप: | Artigo |
| भाषा: | Inglês |
| प्रकाशित: |
AME Publishing Company
2019
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| विषय: | |
| ऑनलाइन पहुंच: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6642929/ https://ncbi.nlm.nih.gov/pubmed/31392202 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/atm.2019.05.56 |
| टैग : |
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