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New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy

Over the past three decades the lysosomal storage diseases have served as model for rare disease treatment development. While these efforts have led to considerable success, important challenges remain. For example, no treatments are currently approved for nearly two thirds of all lysosomal diseases...

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Publicado en:Orphanet J Rare Dis
Autores principales: Schuchman, Edward H., Ledesma, Maria D., Simonaro, Calogera M.
Formato: Artigo
Lenguaje:Inglês
Publicado: BioMed Central 2021
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7992818/
https://ncbi.nlm.nih.gov/pubmed/33766102
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01779-4
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