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Targeted therapies for congenital myasthenic syndromes: systematic review and steps towards a treatabolome
Despite recent scientific advances, most rare genetic diseases — including most neuromuscular diseases — do not currently have curative gene-based therapies available. However, in some cases, such as vitamin, cofactor or enzyme deficiencies, channelopathies and disorders of the neuromuscular junctio...
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| Veröffentlicht in: | Emerg Top Life Sci |
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| Hauptverfasser: | , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Portland Press Ltd.
2019
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6436731/ https://ncbi.nlm.nih.gov/pubmed/30931400 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/ETLS20180100 |
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