How close are we to therapies for Sanfilippo disease?

Lidia Gaffke , Karolina Pierzynowska , Ewa Piotrowska , Grzegorz Węgrzyn

Abstract

n/a
Author Lidia Gaffke KBM
Lidia Gaffke,,
- Department of Molecular Biology
, Karolina Pierzynowska KBM
Karolina Pierzynowska,,
- Department of Molecular Biology
, Ewa Piotrowska KBM
Ewa Piotrowska,,
- Department of Molecular Biology
, Grzegorz Węgrzyn KBM
Grzegorz Węgrzyn,,
- Department of Molecular Biology
Journal seriesMetabolic Brain Disease, ISSN 0885-7490
Issue year2018
Vol33
No1
Pages1-10
Publication size in sheets0.5
Keywords in EnglishSanfilippo disease, neurodegeneration, lysosomal storage, enzyme replacement therapy, gene therapy, substrate reduction therapy
DOIDOI:10.1007/s11011-017-0111-4
URL https://link.springer.com/content/pdf/10.1007%2Fs11011-017-0111-4.pdf
Languageen angielski
LicenseOther; published final; Uznanie Autorstwa (CC-BY); with publication
Score (nominal)25
ScoreMinisterial score = 20.0, 20-12-2017, ArticleFromJournal
Ministerial score (2013-2016) = 25.0, 20-12-2017, ArticleFromJournal
Publication indicators WoS Impact Factor: 2016 = 2.297 (2) - 2016=2.397 (5)
Citation count*0
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