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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Detalles Bibliográficos
Autores principales: Thompson, Rachel, Bonne, Gisèle, Missier, Paolo, Lochmüller, Hanns
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Portland Press Ltd. 2019
Materias:
Review Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6436731/
https://www.ncbi.nlm.nih.gov/pubmed/30931400
http://dx.doi.org/10.1042/ETLS20180100

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6436731/
https://www.ncbi.nlm.nih.gov/pubmed/30931400
http://dx.doi.org/10.1042/ETLS20180100

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