The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase.

Autor: Fog CK; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Zago P; Regional Coordinator Centre for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy., Malini E; Regional Coordinator Centre for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy., Solanko LM; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Peruzzo P; Regional Coordinator Centre for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy., Bornaes C; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Magnoni R; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Mehmedbasic A; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Petersen NHT; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark., Bembi B; Regional Coordinator Centre for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy., Aerts JFMG; Leiden Institute of Chemistry, Leiden University, Leiden, the Netherlands., Dardis A; Regional Coordinator Centre for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy., Kirkegaard T; Orphazyme A/S, Ole Maaloes vej 3, DK-2200 Copenhagen, Denmark. Electronic address: TKJ@orphazyme.com.

Publikováno v: EBioMedicine [EBioMedicine] 2018 Dec; Vol. 38, pp. 142-153. Date of Electronic Publication: 2018 Nov 27.