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BIO12 Impact of Organelle Failure to NPC-C

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mbeds&utm_source=link” target=”_blank” rel=”noopener”>BIO12 Niemann Pick C by Azar Malikzada

Works Cited

Pagon, Roberta A. GeneReviews. University of Washington, Seattle, 2020.

MedlinePlus [Internet]. Bethesda (MD): National Library of Medicine (US); [updated 2020 Jun 24]. Niemann-Pick disease; [updated 2015 Jan 01; cited 2023 Apr 07]. Available from: https://medlineplus.gov/genetics/condition/niemann-pick-disease/

MedlinePlus [Internet]. Bethesda (MD): National Library of Medicine (US); [updated 2020 Jun 24]. Miglustat; [updated 2018 Aug 15; cited 2023 Apr 07]. Available from: https://medlineplus.gov/druginfo/meds/a604015.html

Bianconi, Simona E., et al. “Evaluation of Age of Death in Niemann-Pick Disease, Type C: Utility of Disease Support Group Websites to Understand Natural History.” Molecular Genetics and Metabolism, vol. 126, no. 4, 2019, pp. 466–469., https://doi.org/10.1016/j.ymgme.2019.02.004.

Patterson, Marc C, et al. “Miglustat for Treatment of Niemann-Pick C Disease: A Randomised Controlled Study.” The Lancet Neurology, vol. 6, no. 9, 2007, pp. 765–772., https://doi.org/10.1016/s1474-4422(07)70194-1.

Kirkegaard, Thomas, et al. “Heat Shock Protein–Based Therapy as a Potential Candidate for Treating the Sphingolipidoses.” Science Translational Medicine, vol. 8, no. 355, 2016, https://doi.org/10.1126/scitranslmed.aad9823.

Pineda, Mercè, et al. “Miglustat in Niemann-Pick Disease Type C Patients: A Review.” Orphanet Journal of Rare Diseases, vol. 13, no. 1, 2018, https://doi.org/10.1186/s13023-018-0844-0.

College of Medicine, Albert Einstein, director. YouTube, YouTube, 29 Apr. 2014, https://www.youtube.com/watch?v=EE9kh6OTTSY&ab_channel=AlbertEinsteinCollegeofMedicine. Accessed 7 Apr. 2023.

Flinton, Jill. “What You See and What You Don’t: Diagnosing Niemann Pick, Type C.” Complex Child, 16 Jan. 2018, https://complexchild.org/articles/2011-articles/december/diagnosing-niemann-pick/.

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