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Acid ceramidase deficiency: Farber disease and SMA-PME

Acid ceramidase (ACDase) deficiency is a spectrum of disorders that includes a rare lysosomal storage disorder called Farber disease (FD) and a rare epileptic disorder called spinal muscular atrophy with progressive myoclonic epilepsy (SMA-PME). Both disorders are caused by mutations in the ASAH1 ge...

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Bibliografiske detaljer
Udgivet i:Orphanet J Rare Dis
Main Authors: Yu, Fabian P. S., Amintas, Samuel, Levade, Thierry, Medin, Jeffrey A.
Format: Artigo
Sprog:Inglês
Udgivet: BioMed Central 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6053731/
https://ncbi.nlm.nih.gov/pubmed/30029679
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0845-z
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