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Page 1
Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
J Inherit Metab Dis. 2021 Nov;44(6):1463-1480. doi: 10.1002/jimd.12428. Epub 2021 Sep 7.
J Inherit Metab Dis. 2021.
PMID: 34418116
Free PMC article.
Clinical Trial.
Lysosomal storage diseases and the heat shock response: convergences and therapeutic opportunities.
Ingemann L, Kirkegaard T.
Ingemann L, et al.
J Lipid Res. 2014 Nov;55(11):2198-210. doi: 10.1194/jlr.R048090. Epub 2014 May 16.
J Lipid Res. 2014.
PMID: 24837749
Free PMC article.
Review.
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Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.
Mengel E, Bembi B, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Symonds T, Hudgens S, Patterson MC, Guldberg C, Ingemann L, Petersen NHT, Kirkegaard T, Í Dali C.
Mengel E, et al. Among authors: ingemann l.
Orphanet J Rare Dis. 2020 Nov 23;15(1):328. doi: 10.1186/s13023-020-01616-0.
Orphanet J Rare Dis. 2020.
PMID: 33228797
Free PMC article.
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Correction to: Clinical disease progression and biomarkers in Niemann-Pick disease type C: a prospective cohort study.
Mengel E, Bembi B, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Symonds T, Hudgens S, Patterson MC, Guldberg C, Ingemann L, Petersen NHT, Kirkegaard T, Dali CÍ.
Mengel E, et al. Among authors: ingemann l.
Orphanet J Rare Dis. 2021 Jun 1;16(1):246. doi: 10.1186/s13023-021-01855-9.
Orphanet J Rare Dis. 2021.
PMID: 34074315
Free PMC article.
No abstract available.
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Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.
Kirkegaard T, Gray J, Priestman DA, Wallom KL, Atkins J, Olsen OD, Klein A, Drndarski S, Petersen NH, Ingemann L, Smith DA, Morris L, Bornæs C, Jørgensen SH, Williams I, Hinsby A, Arenz C, Begley D, Jäättelä M, Platt FM.
Kirkegaard T, et al. Among authors: ingemann l.
Sci Transl Med. 2016 Sep 7;8(355):355ra118. doi: 10.1126/scitranslmed.aad9823.
Sci Transl Med. 2016.
PMID: 27605553
Free PMC article.
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