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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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Foilsithe in:Orphanet J Rare Dis
Main Authors: Schuchman, Edward H., Ledesma, Maria D., Simonaro, Calogera M.
Formáid: Artigo
Teanga:Inglês
Foilsithe: BioMed Central 2021
Ábhair:
Rochtain Ar Líne: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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