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Review

Pharmacological treatment of nephropathic cystinosis with cysteamine

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Pages 2255-2262 | Published online: 25 Feb 2005

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Enriko Klootwijk, Stephanie Dufek, Naomi Issler, Detlef Bockenhauer & Robert Kleta. (2017) Pathophysiology, current treatments and future targets in hereditary forms of renal Fanconi syndrome. Expert Opinion on Orphan Drugs 5:1, pages 45-54.
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Gregory M Pastores & Natalie L Barnett. (2005) Current and emerging therapies for the lysosomal storage disorders. Expert Opinion on Emerging Drugs 10:4, pages 891-902.
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Articles from other publishers (59)

Brian K. Lee & Christie P. Thomas. (2024) Genetic testing in the evaluation of recipient candidates and living kidney donors. Current Opinion in Nephrology & Hypertension 33:1, pages 4-12.
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Anna Luiza Braga Albuquerque, Rafael dos Santos Borges, Ana Flávia Conegundes, Erika Emmylaine dos Santos, Frederico Moreira Man Fu, Clara Tavares Araujo, Pedro Alves Soares Vaz de Castro & Ana Cristina Simões e Silva. (2023) Inherited Fanconi syndrome. World Journal of Pediatrics 19:7, pages 619-634.
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Leonie Keidel, Katharina Hohenfellner, Benedikt Schworm, Siegfried Priglinger, Nikolaus Luft & Claudia Priglinger. (2023) Spectral domain optical coherence tomography-based retinochoroidal cystine crystal score: a window into infantile nephropathic cystinosis. British Journal of Ophthalmology 107:2, pages 234-241.
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Trevor F. Killeen, Sarah L. Elfering, Samy M. Riad, Michael A. Linden, Ethan Y. Leng, Raja Kandaswamy, Sarah J. Kizilbash, Blanche M. Chavers & James V. Harmon. (2022) Nephropathic cystinosis in a kidney transplant recipient: A mesenteric lymph node demonstrates positive birefringent crystals. Human Pathology Reports 29, pages 300661.
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Li Zhou, Tiejian Li, Ying Sun, Hongwu Tian, Cunxiu Gao, Chunli Liu, Lingli Kong, Guimin Zhang & Tiesheng Shi. (2020) Mechanistic scrutiny of the oxidations of thiol‐containing drugs cysteamine and d ‐penicillamine by cis ‐diamminetetrachloroplatinum(IV) . International Journal of Chemical Kinetics 53:4, pages 548-557.
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Katharina Hohenfellner & Jens Deerberg-Wittram. (2020) Coordinated, Cost-effective Care for Rare Disease: The Cystinosis Outpatient Consultation Program at RoMed. NEJM Catalyst 1:4.
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Angelina Magreni Dixon, Courtney Styres & Caroline Straatmann. (2020) An Infant With Vomiting, Constipation, and Weight Loss. Clinical Pediatrics 59:6, pages 629-632.
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Ivana Palucci, Giuseppe Maulucci, Flavio De Maio, Michela Sali, Alessandra Romagnoli, Linda Petrone, Gian Maria Fimia, Maurizio Sanguinetti, Delia Goletti, Marco De Spirito, Mauro Piacentini & Giovanni Delogu. (2020) Inhibition of Transglutaminase 2 as a Potential Host-Directed Therapy Against Mycobacterium tuberculosis. Frontiers in Immunology 10.
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Hongnan Liu, Miaomiao Bai, Bie Tan, Kang Xu, Rong Yu, Ruilin Huang & Yulong Yin. (2019) Influence of supplemented coated-cysteamine on morphology, apoptosis and oxidative stress status of gastrointestinal tract. BMC Veterinary Research 15:1.
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F. Cicchetti, L.S. David, A. Siddu & H.L. Denis. (2019) Cysteamine as a novel disease-modifying compound for Parkinson's disease: Over a decade of research supporting a clinical trial. Neurobiology of Disease 130, pages 104530.
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John W. Foreman. (2019) Fanconi Syndrome. Pediatric Clinics of North America 66:1, pages 159-167.
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Danielle Armas, Robert J. Holt, Nils F. Confer, Gregg C. Checani, Mohammad Obaidi, Yuli Xie & Meg Brannagan. (2018) A Phase 1 Pharmacokinetic Study of Cysteamine Bitartrate Delayed-Release Capsules Following Oral Administration with Orange Juice, Water, or Omeprazole in Cystinosis. Advances in Therapy 35:2, pages 199-209.
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Katherine A. Lyseng-Williamson. (2017) Cystadrops® (cysteamine hydrochloride 0.55% viscous eye-drops solution) in treating corneal cystine crystal deposits in patients with cystinosis: a profile of its use. Drugs & Therapy Perspectives 33:5, pages 195-201.
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Neda Moradin, Sabrina Torre, Susan Gauthier, Mifong Tam, Jalal Hawari, Kirsten Vandercruyssen, Bart De Spiegeleer, Anny Fortin, Mary M. Stevenson & Philippe Gros. (2016) Cysteamine broadly improves the anti-plasmodial activity of artemisinins against murine blood stage and cerebral malaria. Malaria Journal 15:1.
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Mike Kalogiannis, E. James Delikatny & Thomas M. Jeitner. (2016) Serotonin as a putative scavenger of hypohalous acid in the brain. Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 1862:4, pages 651-661.
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Allison Berryhill, Suvarna Bhamre, Abanti Chaudhuri, Waldo Concepcion & Paul C. Grimm. (2015) Cysteamine in renal transplantation: A report of two patients with nephropathic cystinosis and the successful re‐initiation of cysteamine therapy during the immediate post‐transplant period. Pediatric Transplantation 20:1, pages 141-145.
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Raymond Quigley & Matthias T. F. Wolf. 2016. Pediatric Nephrology. Pediatric Nephrology 1273 1306 .
John Foreman. (2015) Genetic Diseases of the Kidney. The Open Urology & Nephrology Journal 8:1, pages 136-147.
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Barbara McKenzie, Graeme Kay, Kerr H. Matthews, Rachel M. Knott & Donald Cairns. (2015) The hen’s egg chorioallantoic membrane (HET-CAM) test to predict the ophthalmic irritation potential of a cysteamine-containing gel: Quantification using Photoshop® and ImageJ. International Journal of Pharmaceutics 490:1-2, pages 1-8.
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Galina Nesterova, Caitlyn Williams, Isa Bernardini & William A. Gahl. (2014) Cystinosis: renal glomerular and renal tubular function in relation to compliance with cystine-depleting therapy. Pediatric Nephrology 30:6, pages 945-951.
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G. Ariceta, E. Lara, J. A. Camacho, F. Oppenheimer, J. Vara, F. Santos, M. A. Munoz, C. Cantarell, M. Gil Calvo, R. Romero, B. Valenciano, V. Garcia-Nieto, M. J. Sanahuja, J. Crespo, M. L. Justa, A. Urisarri, R. Bedoya, A. Bueno, A. Daza, J. Bravo, F. Llamas & L. A. Jimenez del Cerro. (2014) Cysteamine (Cystagon(R)) adherence in patients with cystinosis in Spain: successful in children and a challenge in adolescents and adults. Nephrology Dialysis Transplantation 30:3, pages 475-480.
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Pierre Kaminsky & Olivier Lidove. (2014) Stratégies thérapeutiques actuelles dans les maladies lysosomales. La Presse Médicale 43:11, pages 1174-1184.
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Philip J Brooks, Danilo A Tagle & Steve Groft. (2014) Expanding rare disease drug trials based on shared molecular etiology. Nature Biotechnology 32:6, pages 515-518.
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Raymond Quigley & Matthias T. F. Wolf. 2014. Pediatric Nephrology. Pediatric Nephrology 1 40 .
M. Alfadhel, K. Al-Thihli, H. Moubayed, W. Eyaid & M. Al-Jeraisy. (2013) Drug treatment of inborn errors of metabolism: a systematic review. Archives of Disease in Childhood 98:6, pages 454-461.
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C. N. Harvey-Woodworth. (2013) Dimethylsulphidemia: the significance of dimethyl sulphide in extra-oral, blood borne halitosis. British Dental Journal 214:7, pages E20-E20.
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Ranjan Dohil & Patrice Rioux. (2013) Pharmacokinetic Studies of Cysteamine Bitartrate Delayed‐Release. Clinical Pharmacology in Drug Development 2:2, pages 178-185.
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Galina Nesterova & William A. Gahl. (2012) Cystinosis: the evolution of a treatable disease. Pediatric Nephrology 28:1, pages 51-59.
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Dimitrios Tsikas, Christopher E. Evans, Travis T. Denton, Anja Mitschke, Frank-Mathias Gutzki, John T. Pinto, Tetyana Khomenko, Sandor Szabo & Arthur J.L. Cooper. (2012) Stable isotope gas chromatography–tandem mass spectrometry determination of aminoethylcysteine ketimine decarboxylated dimer in biological samples. Analytical Biochemistry 430:1, pages 4-15.
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Ranjan Dohil, Betty L. Cabrera, Jon Gangoiti & Patrice Rioux. (2013) The Effect of Food on Cysteamine Bitartrate Absorption in Healthy Participants. Clinical Pharmacology in Drug Development 1:4, pages 170-174.
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Barbara Buchan, Graeme Kay, Kerr H. Matthews & Donald Cairns. (2012) Suppository formulations as a potential treatment for nephropathic cystinosis. Journal of Pharmaceutical Sciences 101:10, pages 3729-3738.
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Ranjan Dohil, Alison Carrigg & Robert Newbury. (2012) A Potential New Method to Estimate Tissue Cystine Content in Nephropathic Cystinosis. The Journal of Pediatrics 161:3, pages 531-535.e1.
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Stephen Niemiec, Angela Ballantyne & Doris A. Trauner. (2012) Cognition in nephropathic cystinosis: Pattern of expression in heterozygous carriers. American Journal of Medical Genetics Part A 158A:8, pages 1902-1908.
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Toshio Fujisawa, Benjamin Rubin, Akiko Suzuki, Prabhudas S. Patel, William A. Gahl, Bharat H. Joshi & Raj K. Puri. (2012) Cysteamine Suppresses Invasion, Metastasis and Prolongs Survival by Inhibiting Matrix Metalloproteinases in a Mouse Model of Human Pancreatic Cancer. PLoS ONE 7:4, pages e34437.
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Alain Bonnardeaux & Daniel G. Bichet. 2012. Brenner and Rector's The Kidney. Brenner and Rector's The Kidney 1584 1625 .
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Guangmang Liu, Yi Wang, Zhisheng Wang, Jingyi Cai, Xingzuo Lv & Anguo Zhou. (2011) Metabolomic studies on the biochemical profile of urine from rats with acute cysteamine supplementation. Metabolomics 7:4, pages 536-541.
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Guangmang Liu, Yi Wang, Zhisheng Wang, Jingyi Cai, Xingzuo Lv & Anguo Zhou. (2011) Nuclear Magnetic Resonance (NMR)-Based Metabolomic Studies on Urine and Serum Biochemical Profiles after Chronic Cysteamine Supplementation in Rats. Journal of Agricultural and Food Chemistry 59:10, pages 5572-5578.
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Gregory M. Pastores. (2010) Review: Therapeutic approaches for lysosomal storage diseases. Therapeutic Advances in Endocrinology and Metabolism 1:4, pages 177-188.
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S. Terryn, O. Devuyst & C. Antignac. (2010) Cell therapy for cystinosis. Nephrology Dialysis Transplantation 25:7, pages 2103-2106.
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Robert Kleta. (2006) A deeper look into cysteamine absorption for the treatment of cystinosis. The Journal of Pediatrics 148:6, pages 718-719.
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