Erratum for Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders. [Int J Mol Sci. 2016]n/a.info:eu-repo/semantics/publishedVersio

Alves, Sandra

Coutinho, Maria Francisca

Santos, Juliana Inês

PubMed

Maria Francisca Coutinho

Juliana Inês Santos

Sandra Alves

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Coutinho et al. Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders. Int. J. Mol. Sci. 2016, 17, 1065

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 International Journal of Molecular SciencesCorrectionCoutinho et al. Less Is More: Substrate ReductionTherapy for Lysosomal Storage Disorders.Int. J. Mol. Sci. 2016, 17, 1065Maria Francisca Coutinho *, Juliana Inês Santos and Sandra AlvesDepartment of Human Genetics, Research and Development Unit, National HealthInstitute Doutor Ricardo Jorge, Rua Alexandre Herculano, 321 4000-055 Porto, Portugal;juliana.santos@insa.min-saude.pt (J.I.S.); sandra.alves@insa.min-saude.pt (S.A.)* Correspondence: francisca_coutinho@yahoo.com or francisca.coutinho@insa.min-saude.pt;Tel.: +351-223-401-113Academic Editor: Ritva TikkanenReceived: 26 October 2016; Accepted: 21 December 2016; Published: 17 January 2017The authors wish to make a change to their published paper [1] and add novel references.On page 7, second paragraph, the authors stated that “the recommended dose for this oral drug[eliglustat tartrate] is 100 mg, three times a day”. Nevertheless, this dose regimen refers to miglustat,the other SRT drug currently approved for type 1 Gaucher disease. The management recommendationsare different for eliglustat. Eliglustat is a CYP2D6 and CYP3A substrate. Thus, its co-administrationwith drugs that inhibit CYP2D6 and CYP3A may significantly increase the exposure to eliglustat andresult in prolongation of the PR, QTc, and/or QRS cardiac interval, which could result in cardiacarrhythmias [2]. In brief, the recommended dose of 84 mg eliglustat twice daily for extensive andintermediate metabolisers is reduced to 84 mg once daily in poor metabolisers. Still, it is important tonotice that, because of its genetically determined metabolism in the liver, eliglustat requires individualadaptation of the dose and careful supervision of concomitant medications [3].On page 10, the authors refer to an in vivo study using MPS II mice, where a 10-week genisteintreatment resulted in decreased GAG deposits in brain. An important reference, however, has beeninadvertently omitted in the appended review. In 2009, Malinowska and co-workers [4] demonstrateddecreased lysosomal storage in peripheral tissues of a mouse model of Sanfilippo syndrome B(Mucopolysaccharidosis IIIB) treated with genistein. Soon after this first study, the same team providedproof of concept for that isoflavone working as an SRT in the brain of the same mouse model bydemonstrating improved synaptic vesicle protein expression and secondary storage in the cerebralcortex [5]. Considering those results, a phase III double blinded, randomised, placebo controlledclinical trial of high dose oral genistein aglycone in patients with Sanfilippo syndrome was designed,and is currently ongoing in Manchester [6]. Even though its outcomes have not been published to date,it has been widely reported in the field and at MPS meetings.The authors apologize for any inconvenience. The manuscript will be updated, and the originalwill remain online at the article webpage.References1. Coutinho, M.F.; Santos, J.I.; Alves, S. Less is more: Substrate reduction therapy for lysosomal storagedisorders. Int. J. Mol. Sci. 2016, 17, 1065. [CrossRef] [PubMed]2. CerdelgaTM. Highlights of Prescribing Information. Available online: http://www.cerdelga.com/pdf/cerdelga_prescribing_information.pdf (accessed on 26 October 2016).3. Belmatoug, N.; Di Rocco, M.; Fraga, C.; Giraldo, P.; Hughes, D.; Lukina, E.; Maison-Blanche, P.; Merkel, M.;Niederau, C.; Plckinger, U.; et al. Management and monitoring recommendations for the use of eliglustat inadults with type 1 gaucher disease in Europe. Eur. J. Intern. Med. 2016. [CrossRef] [PubMed]Int. J. Mol. Sci. 2017, 18, 178; doi:10.3390/ijms18010178 www.mdpi.com/journal/ijmsInt. J. Mol. Sci. 2017, 18, 178 2 of 24. Malinowska, M.; Wilkinson, F.L.; Bennett, W.; Langford-Smith, K.J.; O’Leary, H.A.; Jakobkiewicz-Banecka, J.;Wynn, R.; Wraith, J.E.; Wegrzyn, G.; Bigger, B.W. Genistein reduces lysosomal storage in peripheral tissuesof mucopolysaccharide IIIB mice. Mol. Genet. Metab. 2009, 98, 235–242. [CrossRef] [PubMed]5. Malinowska, M.; Wilkinson, F.L.; Langford-Smith, K.J.; Langford-Smith, A.; Brown, J.R.; Crawford, B.E.;Vanier, M.T.; Grynkiewicz, G.; Wynn, R.F.; Wraith, J.E.; et al. Genistein improves neuropathology and correctsbehaviour in a mouse model of neurodegenerative metabolic disease. PLoS ONE 2010, 5, e14192. [CrossRef][PubMed]6. The European Union Clinical Trials. High Dose Genistein in Sanfilippo Syndrome. Available online:https://www.clinicaltrialsregister.eu/ctr-search/trial/2013-001479-18/GB (accessed on 26 October 2016).© 2017 by the authors; licensee MDPI, Basel, Switzerland. This article is an open accessarticle distributed under the terms and conditions of the Creative Commons Attribution(CC-BY) license (http://creativecommons.org/licenses/by/4.0/).

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Coutinho et al. Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders. Int. J. Mol. Sci. 2016, 17, 1065

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