2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1
Open accessAbstract
The rare, chronic, autosomal-recessive lysosomal storage disease Niemann-Pick disease type C1 (NPC1) is characterized by progressively debilitating and ultimately fatal neurological manifestations. There is an urgent need for disease-modifying therapies that address NPC1 neurological pathophysiology, and passage through the blood-brain barrier represents an important consideration for novel NPC1 drugs. Animal investigations of 2-hydroxypropyl-β-cyclodextrins (HPβCD) in NPC1 in mice demonstrated that HPβCD does not cross the blood-brain barrier in significant amounts but suggested a potential for these complex oligosaccharides to moderately impact CNS manifestations when administered subcutaneously or intraperitoneally at very high doses; however, safety concerns regarding pulmonary toxicity were raised. Subsequent NPC1 investigations in cats demonstrated far greater HPβCD efficacy at much lower doses when the drug was administered directly to the CNS. Based on this, a phase 1/2a clinical trial was initiated with intrathecal administration of a specific, wellcharacterized mixture of HPβCD, with a tightly controlled molar substitution specification and a defined molecular "fingerprint" of the different species. The findings were very encouraging and a phase 2b/3 clinical trial has completed enrollment and is underway. In addition, phase 1 clinical studies utilizing high-dose intravenous administration of a different HPβCD are currently recruiting. Independent studies are needed for each product to satisfactorily address questions of safety, efficacy, dosing, and route of administration. The outcomes cannot be assumed to be translatable between HPβCD products and/or routes of administration.
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Cyclodextrins can be used for the preparation of formulations able to cross the blood-brain barrier, administered through nasal or oral/parenteral routes.
“For instance, it is known that cyclodextrins can be used for the preparation of formulations able to cross the BBB, administered through nasal or oral/parenteral routes [16,23,80,81].”
From Nanoemulsions for “Nose-to-Brain” Drug Delivery (2019) · cites this paper as [80]
The Introduction (page unavailable) says cyclodextrins are used as excipients in drug formulations, supporting that part of the claim. It does not say those formulations cross the BBB by nasal, oral, or parenteral administration. Considerations and Future Perspectives (page unavailable) instead says the HPβCD studied does not readily cross the BBB in therapeutically relevant amounts. (LLM, conf=0.85)
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Cites
Drug Transport across the Blood–Brain Barrier
Pardridge, W. M. (2012) · Journal of Cerebral Blood Flow & Metabolism · doi:10.1038/jcbfm.2012.126 · Cited by 1,994 · see citing works
Vanier, M. T. (2010) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-5-16 · Cited by 1,222 · see citing works
Cyclodextrins in drug delivery
Loftsson, Þ., Jarho, P., Másson, M., & Järvinen, T. (2005) · Expert Opinion on Drug Delivery · doi:10.1517/17425247.2.1.335 · Cited by 797 · see citing works
Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update
Patterson, M. C., Hendriksz, C. J., Walterfang, M., Sedel, F., Vanier, M. T., & Wijburg, F. A. (2012) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2012.03.012 · Cited by 473 · see citing works
Davidson, C. D., Ali, N. F., Micsenyi, M. C., Stephney, G. W., Renault, S., Dobrenis, K., Ory, D. S., Vanier, M. T., & Walkley, S. U. (2009) · PLoS ONE · doi:10.1371/journal.pone.0006951 · Cited by 449 · see citing works
Liu, B., Turley, S. D., Burns, D. K., Miller, A. M., Repa, J. J., & Dietschy, J. M. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0810895106 · Cited by 376 · see citing works
Ory, D. S., Ottinger, E. A., Farhat, N. Y., King, K. A., Jiang, X., Weissfeld, L. A., Berry‐Kravis, E., Davidson, C. D., Bianconi, S. E., Keener, L. A., et al. (2017) · The Lancet · doi:10.1016/s0140-6736(17)31465-4 · Cited by 318 · see citing works
Hydroxypropyl-β-cyclodextrin: preparation and characterization; effects on solubility of drugs
Pitha, J., Milecki, J., Fales, H. M., Pannell, L. K., & Uekama, K. (1986) · International Journal of Pharmaceutics · doi:10.1016/0378-5173(86)90201-2 · Cited by 304 · see citing works
Complex lipid trafficking in Niemann‐Pick disease type C
Vanier, M. T. (2014) · Journal of Inherited Metabolic Disease · doi:10.1007/s10545-014-9794-4 · Cited by 241 · see citing works
Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease
Camargo, F. D., Erickson, R. P., Garver, W. S., Hossain, G. S., Carbone, P. N., Heidenreich, R. A., & Blanchard, J. D. (2001) · Life Sciences · doi:10.1016/s0024-3205(01)01384-4 · Cited by 202 · see citing works
Vite, C. H., Bagel, J. H., Swain, G. P., Prociuk, M. L., Sikora, T. U., Stein, V. M., O’Donnell, P., Ruane, T., Ward, S. E., Crooks, A. V., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.3010101 · Cited by 202 · see citing works
Intrathecal delivery of protein therapeutics to the brain: a critical reassessment.
Calias, P., Banks, W., Begley, D., Scarpa, M., & Dickson, P. (2014) · Pharmacology and Therapeutics · doi:10.1016/j.pharmthera.2014.05.009 · Cited by 157 · see citing works
Self-citationAqul, A. A., Liu, B., Ramírez, C. M., Pieper, A. A., Estill, S. J., Burns, D. K., Liu, B., Repa, J. J., Turley, S. D., & Dietschy, J. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.1317-11.2011 · Cited by 152 · see citing works
Effects of cyclodextrin in two patients with Niemann–Pick Type C disease
Matsuo, M., Togawa, M., Hirabaru, K., Mochinaga, S., Narita, A., Adachi, M., Egashira, M., Irie, T., & Ohno, K. (2012) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2012.11.005 · Cited by 139 · see citing works
Ottinger, E., Kao, M., Carrillo-Carrasco, N., Yanjanin, N., Shankar, R., Janssen, M., Brewster, M., Scott, I., Xu, X., Cradock, J., et al. (2014) · Current Topics in Medicinal Chemistry · doi:10.2174/1568026613666131127160118 · Cited by 121 · see citing works
Ward, S. E., O’Donnell, P., Fernandez, S., & Vite, C. H. (2010) · Pediatric Research · doi:10.1203/pdr.0b013e3181df4623 · Cited by 112 · see citing works
Cyclodextrins, Blood–Brain Barrier, and Treatment of Neurological Diseases
Vecsernyés, M., Fenyvesi, F., Bácskay, I., Deli, M. A., Szente, L., & Fenyvesi, É. (2014) · Archives of Medical Research · doi:10.1016/j.arcmed.2014.11.020 · Cited by 102 · see citing works
Stein, V. M., Crooks, A. V., Ding, W., Prociuk, M. L., O’Donnell, P., Bryan, C., Sikora, T. U., Dingemanse, J., Vanier, M. T., Walkley, S. U., et al. (2012) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/nen.0b013e31825414a6 · Cited by 94 · see citing works
Efficacy and ototoxicity of different cyclodextrins in Niemann–Pick C disease
Davidson, C. D., Fishman, Y. I., Puskás, I., Szemán, J., Sohajda, T., McCauliff, L. A., Sikora, J., Storch, J., Vanier, M. T., Szente, L., et al. (2016) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.306 · Cited by 89 · see citing works
Pontikis, C., Davidson, C. D., Walkley, S. U., Platt, F. M., & Begley, D. J. (2013) · Journal of Inherited Metabolic Disease · doi:10.1007/s10545-012-9583-x · Cited by 83 · see citing works
Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann–Pick C1
Maarup, T. J., Chen, A. H., Porter, F. D., Farhat, N. Y., Ory, D. S., Sidhu, R., Jiang, X., & Dickson, P. (2015) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2015.07.001 · Cited by 82 · see citing works
Ramirez, C. M., Liu, B., Aqul, A. A., Taylor, A. M., Repa, J. J., Turley, S. D., & Dietschy, J. M. (2011) · Journal of Lipid Research · doi:10.1194/jlr.m013789 · Cited by 76 · see citing works
Vanier, M. T., Platt, F. M., Eden, E. R., & Patterson, M. C. (2022) · doi:10.1002/9781119697312.ch14 · Cited by 76 · see citing works
Tortelli, B. A., Fujiwara, H., Bagel, J. H., Zhang, J., Sidhu, R., Jiang, X., Yanjanin, N. M., Shankar, R. K., Carrillo, N., Heiss, J. D., et al. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu331 · Cited by 54 · see citing works
Developing drugs that can cross the blood-brain barrier: applications to Alzheimer's disease
Banks, W. A. (2008) · BMC Neuroscience · doi:10.1186/1471-2202-9-s3-s2 · Cited by 51 · see citing works
Matsuo, M., Shraishi, K., Wada, K., Ishitsuka, Y., Doi, H., Maeda, M., Mizoguchi, T., Eto, J., Mochinaga, S., Arima, H., et al. (2014) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2014.08.004 · Cited by 50 · see citing works
Auditory Phenotype of Niemann-Pick Disease, Type C1
King, K. A., Gordon-Salant, S. M., Yanjanin, N. M., Zalewski, C. K., Houser, A., Porter, F. D., & Brewer, C. C. (2013) · Ear and Hearing · doi:10.1097/aud.0b013e3182a362b8 · Cited by 50 · see citing works
A Comparative Overview of Prescription Omega-3 Fatty Acid Products.
Ito, M. K. (2015) · PubMed Central · Cited by 49 · see citing works
Vite, C. H., Ding, W., Bryan, C., O’Donnell, P., Cullen, K., Aleman, D. O., Haskins, M. E., & Winkle, T. V. (2008) · Pediatric Research · doi:10.1203/pdr.0b013e318184d2ce · Cited by 40 · see citing works
Lopez, A. M., Terpack, S. J., Posey, K. S., Liu, B., Ramirez, C. M., & Turley, S. D. (2014) · Clinical and Experimental Pharmacology and Physiology · doi:10.1111/1440-1681.12285 · Cited by 38 · see citing works
Alobaidy, H. (2015) · International Journal of Pediatrics · doi:10.1155/2015/816593 · Cited by 38 · see citing works
Yergey, A. L., Blank, P. S., Cologna, S. M., Backlund, P. S., Porter, F. D., & Darling, A. J. (2017) · PLoS ONE · doi:10.1371/journal.pone.0175478 · Cited by 36 · see citing works
Molecular Genetics and Metabolism Reports
(2015) · Molecular Genetics and Metabolism · doi:10.1016/s1096-7192(15)00146-8 · Cited by 36 · see citing works
Diagnostic workup and management of patients with suspected Niemann-Pick type C disease
Papandreou, A., & Gissen, P. (2016) · Therapeutic Advances in Neurological Disorders · doi:10.1177/1756285616635964 · Cited by 22 · see citing works
Use of 2 hydroxypropyl-beta-cyclodextrin therapy in two adult Niemann Pick Type C patients
García-Robles, A. A., Megías‐Vericat, J. E., Fernández-Megía, M. J., Miralles, F. C. P., Briz, E. L., Alcalà-Vicente, C., Galeano, I., Casanova, B., & Poveda, J. L. (2016) · Journal of the Neurological Sciences · doi:10.1016/j.jns.2016.04.048 · Cited by 19 · see citing works
Flagged first, then by citations
1 of the 36 references couldn’t be matched to a known paper.
Cited by
Sorted by citations
Nanoemulsions for “Nose-to-Brain” Drug Delivery
Bonferoni, M. C., Rossi, S., Sandri, G., Ferrari, F., Gavini, E., Rassu, G., & Giunchedi, P. (2019) · Pharmaceutics · doi:10.3390/pharmaceutics11020084 · Cited by 269 · see citing works
Niemann–Pick type C disease: cellular pathology and pharmacotherapy
Wheeler, S., & Sillence, D. J. (2019) · Journal of Neurochemistry · doi:10.1111/jnc.14895 · Cited by 107 · see citing works
Chitosan-Based Nanocarriers for Nose to Brain Delivery
Aderibigbe, B. A., & Naki, T. (2019) · Applied Sciences · doi:10.3390/app9112219 · Cited by 104 · see citing works
Anti-inflammatory potential of quercetin: From chemistry and mechanistic insight to nanoformulations
Aggarwal, D., Chaudhary, M., Mandotra, S. K., Tuli, H. S., Chauhan, R., Joshi, N. C., Kaur, D., Dufossé, L., & Chauhan, A. P. S. (2025) · Current Research in Pharmacology and Drug Discovery · doi:10.1016/j.crphar.2025.100217 · Cited by 76 · see citing works
Lysosomal storage diseases: current therapies and future alternatives
Leal, A. F., Espejo-Mojica, A. J., Sánchez, O. F., Ramírez, C. M., Reyes, L. H., Cruz, J. C., & Alméciga-Díaz, C. J. (2020) · Journal of Molecular Medicine · doi:10.1007/s00109-020-01935-6 · Cited by 65 · see citing works
Versatile Nasal Application of Cyclodextrins: Excipients and/or Actives?
Rassu, G., Sorrenti, M., Catenacci, L., Pavan, B., Ferraro, L., Gavini, E., Bonferoni, M. C., Giunchedi, P., & Dalpiaz, A. (2021) · Pharmaceutics · doi:10.3390/pharmaceutics13081180 · Cited by 51 · see citing works
Cyclic Oligosaccharides as Active Drugs, an Updated Review
Matencio, A., Caldera, F., Cecone, C., López-Nicolás, M., & Trotta, F. (2020) · Pharmaceuticals · doi:10.3390/ph13100281 · Cited by 50 · see citing works
Recent advances in the treatment of Niemann pick disease type C: A mini-review
Matencio, A., Navarro‐Orcajada, S., González-Ramón, A., García‐Carmona, F., & López-Nicolás, M. (2020) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2020.119440 · Cited by 46 · see citing works
Routes for the delivery of insulin to the central nervous system: A comparative review
Rhea, E. M., Salameh, T. S., & Banks, W. A. (2019) · Experimental Neurology · doi:10.1016/j.expneurol.2018.11.007 · Cited by 43 · see citing works
Inherited disorders of lysosomal membrane transporters
Huizing, M., & Gahl, W. A. (2020) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2020.183336 · Cited by 39 · see citing works
Graphene Quantum Dots Alleviate Impaired Functions in Niemann-Pick Disease Type C in Vivo
Kang, I., Yoo, J. M., Kim, D., Kim, J., Cho, M. K., Lee, S. E., Kim, D. J., Lee, B.-C., Lee, J. Y., Kim, J.-J., et al. (2021) · Nano Letters · doi:10.1021/acs.nanolett.0c03741 · Cited by 38 · see citing works
El‐Darzi, N., Mast, N., Petrov, A. M., & Pikuleva, I. A. (2020) · British Journal of Pharmacology · doi:10.1111/bph.15209 · Cited by 30 · see citing works
Myelin Defects in Niemann–Pick Type C Disease: Mechanisms and Possible Therapeutic Perspectives
Bernardo, A., Nuccio, C. D., Visentin, S., Martire, A., Minghetti, L., Popoli, P., & Ferrante, A. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22168858 · Cited by 26 · see citing works
Carradori, D., Chen, H., Werner, B., Shah, A., Leonardi, C., Usuelli, M., Mezzenga, R., Platt, F., & Leroux, J. (2020) · Small · doi:10.1002/smll.202004735 · Cited by 19 · see citing works
Zhou, Y., Takahashi, S., Homma, K., Duan, C., Zheng, J., Cheatham, M. A., & Zheng, J. (2018) · Acta Neuropathologica Communications · doi:10.1186/s40478-018-0599-9 · Cited by 21 · see citing works
Banks, W. (2020) · Current pharmaceutical design · doi:10.2174/138161282601200225102049 · Cited by 7 · see citing works
Banks, W. A., Engelke, K. J., Hansen, K. M., Bullock, K. M., & Calias, P. (2019) · Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.119.260497 · Cited by 15 · see citing works
Self-citationTrencsényi, G., Kis, A., Szabó, J. P., Ráti, Á., Csige, K., Fenyvesi, É., Szente, L., Malanga, M., Méhes, G., Emri, M., et al. (2020) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2019.118954 · Cited by 14 · see citing works
Chen, Y., & Liu, A. (2024) · Polymer · doi:10.1016/j.polymer.2024.127788 · Cited by 9 · see citing works
Moiz, B., Vargas, V. A., Brandon, K. D., Sangha, G. S., Weber, C. M., Li, A., Pepper, T., Walls, M., Qin, A., Hart, S., et al. (2025) · Annals of Biomedical Engineering · doi:10.1007/s10439-025-03841-9 · Cited by 9 · see citing works
Alterations in Proteostasis Mechanisms in Niemann–Pick Type C Disease
Servín-Muñoz, I. V., Ortuño‐Sahagún, D., Griñán‐Ferré, C., Pallàs, M., & González-Castillo, C. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25073806 · Cited by 8 · see citing works
Cyclodextrin-Appended Superparamagnetic Iron Oxide Nanoparticles as Cholesterol-Mopping Agents
Puglisi, A., Bassini, S., & Reimhult, E. (2021) · Frontiers in Chemistry · doi:10.3389/fchem.2021.795598 · Cited by 6 · see citing works
Du, J., Liu, X., Zhang, Y., Han, X., Ma, C., Liu, Y., Guan, L., Qiao, L., & Lin, J. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2021.825425 · Cited by 6 · see citing works
Thiberville, L., Faivre, V., Sizun, C., Dehouck, M., Landry, C., Baati, R., & Tsapis, N. (2025) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2025.125505 · Cited by 5 · see citing works
El‐Darzi, N., Mast, N., Hammer, S. S., Dorweiler, T. F., Busik, J. V., & Pikuleva, I. A. (2022) · Journal of Lipid Research · doi:10.1016/j.jlr.2022.100323 · Cited by 5 · see citing works
The Blood–Brain Barrier and CNS Drug Delivery
Banks, W. A. (2021) · Burger's Medicinal Chemistry and Drug Discovery · doi:10.1002/0471266949.bmc285 · Cited by 5 · see citing works
Suhail, H., Rahman, M. A., Yadab, M. K., Gonawala, S., deCarvalho, A. C., Ewing, J. R., Snyder, J., & Ali, M. M. (2025) · Scientific Reports · doi:10.1038/s41598-025-14731-4 · Cited by 4 · see citing works
Sleep loss induces cholesterol-associated myelin dysfunction
Simayi, R., Ficiarà, E., Faniyan, O. O., Cerdá, A. C., Alaoui, A. A. E., Fiorini, R., Cutignano, A., Piscitelli, F., Maroto, A. S., Santos, A., et al. (2026) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2523438123 · Cited by 5 · see citing works
Serendipitous effects of β-cyclodextrin on murine model of Krabbe disease
Katabuchi, A., Godoy, V., Shil, P., Moser, A. B., & Maegawa, G. H. B. (2018) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2018.03.002 · Cited by 4 · see citing works
Peripheral immune system modulates Purkinje cell degeneration in Niemann–Pick disease type C1
Yasuda, T., Uchiyama, T., Watanabe, N., Ito, N., Nakabayashi, K., Mochizuki, H., & Onodera, M. (2023) · Life Science Alliance · doi:10.26508/lsa.202201881 · Cited by 4 · see citing works
Therapies for Lysosomal Storage Diseases
Desnick, R. J., Astrin, K. H., & Schuchman, E. (2019) · Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics · doi:10.1016/b978-0-12-812536-6.00007-9 · Cited by 1 · see citing works
Termsarasab, P., & Frucht, S. J. (2021) · Elsevier eBooks · doi:10.1016/b978-0-12-820472-6.00146-8 · Cited by 2 · see citing works
Yamada, Y., Ishitsuka, Y., Fukaura, M., Kawata, T., Ishii, A., Shirakawa, A., Sakai, T., Tanaka, M., Kondo, Y., Takeo, T., et al. (2024) · Life Sciences · doi:10.1016/j.lfs.2024.122776 · Cited by 2 · see citing works
Antipova, V., Heimes, D., Seidel, K., Schulz, J., Schmitt, O., Holzmann, C., Rolfs, A., Bidmon, H. J., Román, E. G. D. S., Huesgen, P. F., et al. (2024) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2024.1430790 · Cited by 2 · see citing works
Synthesis and biological evaluation of novel β-cyclodextrin-fluvastatin conjugates
Nicolosi, M., Bellia, F., Giuffrida, M. L., Zimbone, S., Oliveri, V., & Vecchio, G. (2021) · Results in Chemistry · doi:10.1016/j.rechem.2021.100230 · Cited by 1 · see citing works
Just, N. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.07.14.203067
Carradori, D., Chen, H., Werner, B., Shah, A. S., Leonardi, C., Usuelli, M., Mezzenga, R., Platt, F. M., & Leroux, J. (2020) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2020.07.31.230136
The Dilemma of Insulin Delivery into the Brain: A Comprehensive Review
Hatami, H., Askari, V. R., Rahimi, V. B., Hasnain, M. S., & Nayak, A. K. (2024) · Current Drug Delivery · doi:10.2174/0115672018305677240611080910
Synthesis of a Hydroxy-15-Azasterol
Jones, C. A. H., Melancon, B. J., & Lindsley, C. W. (2025) · ACS Omega · doi:10.1021/acsomega.4c09907
Treating glioblastoma with dextrin-based polymers
Bastiancich, C., Molinar, C., Tchoghandjian, A., Cavalli, R., & Scomparin, A. (2025) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2025.2570845
Study of the Complexation of Thiowurtzine with Cyclodextrins
Lobova, N., Aleksandrova, A. V., Mediantsev, E. S., Aleхandrov, A. A., Avakyan, V. G., Titov, S. V., & Vatsadze, S. Z. (2026) · Russian Journal of General Chemistry · doi:10.1134/s1070363225607574
Wuerch, E. C., Baaklini, C. S., Zhang, P., Mørch, M. T., Che, A., D’Mello, C., Ling, C., & Yong, V. W. (2026) · Neurotherapeutics · doi:10.1016/j.neurot.2026.e00943
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West, N. R., & Julia, T. C. W. (2026) · Cell stem cell · doi:10.1016/j.stem.2026.08.007
- Authors
- Calias, P.
- Affiliations
- Co-Founder and Pharmaceutical Development Consultant, Educational Trainers and Consultants, 39 Swains Pond Ave, Melrose, MA 02176, United States
- Corresponding author
- Calias, Pericles
- Venue
- Current Pharmaceutical Design
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2018-02-15
- Volume
- 23
- Issue
- 40
- Pages
- 6231-6238
- ISSN
- 1381-6128 (ISSN-L)
- PMC
- PMC5824462
- OpenAlex
- W2765725508
- Cited by
- 44
- References
- 36
- Citations by year
- 2026: 4 · 2025: 6 · 2024: 5 · 2023: 1 · 2022: 2 · 2021: 7 · 2020: 10 · 2019: 5 · 2018: 4
- FWCI
- 0.85
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Current Pharmaceutical Design (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Health Sciences › Medicine › Pathology and Forensic Medicine › Biomedical Research and Pathophysiology
- Keywords
- Niemann-Pick disease type C1, hydroxypropyl-β-cyclodextrin, blood-brain barrier, lysosomal storage disease, intrathecal administration, brain drug delivery, pulmonary toxicity, drug safety, route of administration, clinical trials
- MeSH terms
- 2-Hydroxypropyl-beta-cyclodextrin, Animals, Blood-Brain Barrier, Disease Models, Animal, Humans, Niemann-Pick Disease, Type C
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