المؤلفون: Giugliani R; Dep Genetics UFRGS, Casa dos Raros, INAGEMP, Med Genet Serv HCPA, and DASA Genomics, Porto Alegre, Brazil. rgiugliani@hcpa.edu.br., Gonzalez-Meneses A; Hospital Universitario Virgen del Rocio, Seville, Spain., Scarpa M; Regional Coordinator Centre for Rare Diseases, University Hospital of Udine, Udine, Italy., Burton B; Feinberg School of Medicine, Northwestern University, Chicago, IL, USA., Wang R; University of California Irvine School of Medicine, Children's Health of Orange County, Orange, CA, USA., Martins E; Centro Hospitalar Universitário de Santo António, Porto, Portugal., Oussoren E; Erasmus University Medical Center Rotterdam, Rotterdam, The Netherlands., Hennermann JB; University Medical Center Mainz, Villa Metabolica, Mainz, Germany., Chabrol B; Centre Hospitalier Universitaire La Timone, Marseille, France., Grant CL; Children's National Hospital, Washington, District of Columbia, USA., Sun A; Seattle Children's Hospital, Seattle, WA, USA., Durand C; Laboratorio Chamoles, Buenos Aires, Argentina., Hetzer J; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Malkus B; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Marsden D; Ultragenyx Pharmaceutical Inc, Novato, CA, USA., Merritt Ii JL; Ultragenyx Pharmaceutical Inc, Novato, CA, USA.

المصدر: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 May 07; Vol. 19 (1), pp. 189. Date of Electronic Publication: 2024 May 07.

نوع المنشور: Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: BioMed Central Country of Publication: England NLM ID: 101266602 Publication Model: Electronic Cited Medium: Internet ISSN: 1750-1172 (Electronic) Linking ISSN: 17501172 NLM ISO Abbreviation: Orphanet J Rare Dis Subsets: MEDLINE

مواضيع طبية MeSH: Mucopolysaccharidosis VII*/drug therapy , Glucuronidase*/therapeutic use , Glucuronidase*/metabolism , Enzyme Replacement Therapy*/methods , Recombinant Proteins*/therapeutic use , Recombinant Proteins*/administration & dosage , Recombinant Proteins*/adverse effects, Humans ; Male ; Child, Preschool ; Female ; Child ; Infant ; Longitudinal Studies ; Adolescent

المؤلفون: Anderson, Julia, Seol, Haeri, Gordish-Dressman, Heather, Hathout, Yetrib, Spurney, Christopher F., McDonald, C., Abresch, R. T., Henricson, E., Cregan, M., Goude, E., Johnson, L., Han, J., Joyce, N., Reedy, D., Viswanathan, V., Chidambaranathan, C., Kumar, S., Lakshmi, V., Reddappa, P., Biggar, D., McAdam, L., Dermody, M., Eliasoph, L., Harris., V., Lee, G., Mah, J., Chiu, A., Haig, T., Harris, M., Sanchez, K., Thannhauser, J., Walker, L., Wright, C., Tulinius, M., Alhander, A., Ekstrom, A., Gustafsson, A., Kroksmark, A., Sterky, U., Wahlgren, L., Thangarajh, M., Birkmeier, M., Kaminski, S., Tadesse, B., Toles, A., Kornberg, A., Ryan, M., Carroll, K., DeValle, K., Kennedy, R., Rodriguez, V., Villano, D., Nevo, Y., Adani, R., Chen-Joseph, L., Simchovitz, E., Yaffe, D., Dubrovsky, A., Andreone, L., Bonaudo, F., Corderi, J., Mesa, L., Marco, P., Levi, L., Clemens, P., Abdel-Hamid, H., Bendixen, R., Bise, C., Craig, A., Karnavas, K., Matthews, C., Niizawa, G., Smith, A., Weimer, J., Connolly, A., Pestronk, A., Florence, J., Christenson, T., Golumbak, P., Lopate, G., Malane, J., Malkus, B., Renna, R., Schierbacker, J., Seiner, C., Wulf, C., Teasley, J., Blair, S., Grillo, B., Jones, K., Monasterio, E., Bertorini, T., Igarashi, M., Barrett-Adair, M., Carter, K., Clift, J., Feliciano, C., Gatlin, B., Holloway, J., Young, R., Webster, R., North, K., Cornett, K., Gabriel, N., Miller, C., Rose, K., Wicks, S., Kolski, H., Chen, L., Kennedy, C., Gorni, K., Beneggi, M., Capone, L., Molteni, A., Morettini, V., Lotze, T., Gupta, A., Knight, A., Lott, B., McNeil, R., Karachunski, P., Day, J., Chambers, G., Dalton, J., Erickson, A., Margolis, M., Marsh, J., Naughton, C., Cnaan, A., Ahmed, M., Arrieta, A., Bartley, N., Brown-Caines, T., Canelos, P., Casper, R., Duong, T., Feng, J., Gordish-Dressman, H., Morgenroth, L., Hu, F., Hunegs, L., Sund, Z., Zimmerman, A., On behalf of the CINRG Investigators

المصدر: Pediatric Cardiology. :1-7

المؤلفون: Malkus, B., O'Mara, A., Sankoh, S.

المصدر: In Value in Health June 2024 27(6) Supplement:S17-S17

المؤلفون: Dress, A, Theodore-Oklota, C, Malkus, B, Egan, S, Umakanth, S, Evans, C

المصدر: In Value in Health December 2022 25(12) Supplement:S402-S402

المؤلفون: Lau HA; NYU Grossman School of Medicine, Department of Neurology, New York, NY, USA. Electronic address: Heather.Lau@nyulangone.org., Viskochil D; University of Utah, Department of Pediatrics, Salt Lake City, UT, USA. Electronic address: Dave.Viskochil@hsc.utah.edu., Tanpaiboon P; Rare Disease Institute, Children's National Health System, Washington, DC, USA. Electronic address: PTanpaib@childrensnational.org., Lopez AG; Hospital Universitario Virgen del Rocío and Universidad de Sevilla, Seville, Spain. Electronic address: antonio.gonzalezmeneses.l.sspa@juntadeandalucia.es., Martins E; Centro Hospitalar Universitário do Porto, Hospital de Santo António, Porto, Portugal. Electronic address: esmeralda.dia@chporto.min-saude.pt., Taylor J; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: JTaylor@ultragenyx.com., Malkus B; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: BMalkus@ultragenyx.com., Zhang L; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: LZhang@ultragenyx.com., Jurecka A; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: ajurecka@gmail.com., Marsden D; Ultragenyx Pharmaceutical Inc., Novato, CA, USA. Electronic address: DMarsden@ultragenyx.com.

المصدر: Molecular genetics and metabolism [Mol Genet Metab] 2022 May; Vol. 136 (1), pp. 28-37. Date of Electronic Publication: 2022 Mar 09.

نوع المنشور: Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Academic Press Country of Publication: United States NLM ID: 9805456 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1096-7206 (Electronic) Linking ISSN: 10967192 NLM ISO Abbreviation: Mol Genet Metab Subsets: MEDLINE

مواضيع طبية MeSH: Mucopolysaccharidosis VII*/therapy, Child ; Enzyme Replacement Therapy ; Glucuronidase ; Glycosaminoglycans/urine ; Hepatomegaly ; Humans ; Hydrolases ; Splenomegaly

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المؤلفون: Scott, R, Best, J, Forder, P, Taskinen, M-R, Simes, J, Barter, P, Keech, A, Colman, P, D'Emden, M, Davis, T, Drury, P, Ehnholm, C, Glasziou, P, Hunt, D, Kesaniemi, YA, Laakso, M, Simes, RJ, Sullivan, D, Whiting, M, Ansquer, J-C, Fraitag, B, Anderson, N, Hankey, G, Lehto, S, Mann, S, Romo, M, Li, LP, Hennekens, C, MacMahon, S, Pocock, S, Tonkin, A, Wilhelmsen, L, Akauola, H, Alford, F, Beinart, I, Bohra, S, Boyages, S, Connor, H, Darnell, D, Davoren, P, Lepre, F, De Looze, F, Duffield, A, Fassett, R, Flack, J, Fulcher, G, Grant, S, Hamwood, S, Harmelin, D, Jackson, R, Jeffries, W, Kamp, M, Kritharides, L, Mahar, L, McCann, V, McIntyre, D, Moses, R, Newnham, H, Nicholson, G, O'Brien, R, Park, K, Petrovsky, N, Phillips, P, Pinn, G, Simmons, D, Stanton, K, Stuckey, B, Sullivan, DR, Suranyi, M, Suthers, M, Tan, Y, Templer, M, Topliss, D, Waites, JH, Watts, G, Welborn, T, Wyndham, R, Haapamaki, H, Kesaniemi, A, Lahtela, J, Levanen, H, Saltevo, J, Sodervik, H, Taskinen, M, Vanhala, M, Baker, J, Burton, A, Dixon, P, Doran, J, Dunn, P, Graham, N, Hamer, A, Hedley, J, Lloyd, J, Manning, P, McPherson, I, Morris, S, Renner, C, Smith, R, Wackrow, M, Young, S, Alard, F, Alcoe, J, Allan, C, Amerena, J, Anderson, R, Arnold, N, Arsov, T, Ashby, D, Atkinson, C, Badhni, L, Balme, M, Barton, D, Batrouney, B, Beare, C, Beattie, T, Beggs, J, Bendall, C, Benz, A, Bond, A, Bradfield, R, Bradshaw, J, Brearley, S, Bruce, D, Burgess, J, Butler, J, Callary, M, Campbell, J, Chambers, K, Chow, J, Chow, S, Ciszek, K, Clifton, P, Clifton-Bligh, P, Clowes, V, Coates, P, Cocks, C, Cole, S, Colquhoun, D, Correcha, M, Costa, B, Coverdale, S, Croft, M, Crowe, J, Dal Sasso, S, Davis, W, Dunn, J, Edwards, S, Elder, R, El-Kaissi, S, Emery, L, England, M, Farouque, O, Fernandez, M, Fitzpatrick, B, Francis, N, Freeman, P, Fuller, A, Gale, D, Gaylard, V, Gillzan, C, Glatthaar, C, Goddard, J, Grange, V, Greenaway, T, Griffin, J, Grogan, A, Guha, S, Gustafson, J, Hamblin, PS, Hannay, T, Hardie, C, Harper, A, Hartl, G, Harvey, A, Havlin, S, Haworth, K, Hay, P, Hay, L, Heenan, B, Hesketh, R, Heyworth, A, Hines, M, Hockings, G, Hodge, A, Hoffman, L, Hoskin, L, Howells, M, Hunt, A, Inder, W, Jackson, D, Jovanovska, A, Kearins, K, Kee, P, Keen, J, Kilpatrick, D, Kindellan, J, Kingston-Ray, M, Kotowicz, M, Lassig, A, Layton, M, Lean, S, Lim, E, Long, F, Lucas, L, Ludeman, D, Ludeman-Robertson, C, Lyall, M, Lynch, L, Maddison, C, Malkus, B, Marangou, A, Margrie, F, Matthiesson, K, Matthiesson, J, Maxwell, S, McCarthy, K, McElduff, A, Mckee, H, McKenzie, J, McLachan, K, McNair, P, Meischke, M, Miller, AMC, Morrison, B, Morton, A, Mossman, W, Mowat, A, Muecke, J, Murie, P, Murray, S, Nadorp, P, Nair, S, Nairn, J, Nankervis, A, Narayan, K, Nattrass, N, Ngui, J, Nicholls, S, Nicholls, V, Nye, JA, Nye, E, O'Neal, D, O'Neill, M, O'Rourke, S, Pearse, J, Pearson, C, Phillips, J, Pittis, L, Playford, D, Porter, L, Portley, R, Powell, M, Preston, C, Pringle, S, Quinn, WA, Raffaele, J, Ramnath, G, Ramsden, J, Richtsteiger, D, Roffe, S, Rosen, S, Ross, G, Ross, Z, Rowe, J, Rumble, D, Ryan, S, Sansom, J, Seymour, C, Shanahan, E, Shelly, S, Shepherd, J, Sherman, G, Siddall, R, Silva, D, Simmons, S, Simpson, R, Sinha, A, Slobodniuk, R, Smith, M, Smith, P, Smith, S, Smith-Orr, V, Snow, J, Socha, L, Stack, T, Steed, K, Steele, K, Stephensen, J, Stevens, P, Stewart, G, Stewart, R, Strakosch, C, Sullivan, M, Sunder, S, Sunderland, J, Tapp, E, Taylor, J, Thorn, D, Tolley, A, Torpy, D, Truran, G, Turner, F, Turner, J, Van de Velde, J, Varley, S, Wallace, J, Walsh, J, Walshe, J, Ward, G, Watson, B, Watson, J, Webb, A, Werner, F, White, E, Whitehouse, A, Whitehouse, N, Wigg, S, Wilkinson, J, Wilmshurst, E, Wilson, D, Wittert, G, Wong, B, Wong, M, Worboys, S, Wright, S, Wu, S, Yarker, J, Yeo, M, Young, K, Youssef, J, Yuen, R, Zeimer, H, Ziffer, RW, Aura, A, Friman, A, Hanninen, J, Henell, J, Hyvarinen, N, Ikonen, M, Itkonen, A, Jappinen, J, Jarva, A, Jerkkola, T, Jokinen, V, Juutilainen, J, Kahkonen, H, Kangas, T, Karttunen, M, Kauranen, P, Kortelainen, S, Koukkunen, H, Kumpulainen, L, Laitinen, T, Laitinen, M, Lehto, R, Leinonen, E, Lindstron-Karjalainen, M, Lumiaho, A, Makela, J, Makinen, K, Mannermaa, L, Mard, T, Miettinen, J, Naatti, V, Paavola, S, Parssinen, N, Ripatti, J, Ruotsalainen, S, Salo, A, Siiskonen, M, Soppela, A, Starck, J, Suonranta, I, Ukkola, L, Valli, K, Virolainen, J, Allan, P, Arnold, W, Bagg, W, Balfour, K, Ball, T, Ballantine, B, Ballantyne, C, Barker, C, Bartley, F, Berry, E, Braatvedt, G, Campbell, A, Clarke, T, Clarke, R, Claydon, A, Clayton, S, Cresswell, P, Cutfield, R, Daffurn, J, Delahunt, J, Dissnayake, A, Eagleton, C, Ferguson, C, Florkowski, C, Fry, D, Giles, P, Gluyas, M, Grant, C, Guile, P, Guolo, M, Hale, P, Hammond, M, Healy, P, Hills, M, Hinge, J, Holland, J, Hyne, B, Ireland, A, Johnstone, A, Jones, S, Kerr, G, Kerr, K, Khant, M, Krebs, J, Law, L, Lydon, B, MacAuley, K, McEwan, R, McGregor, P, McLaren, B, McLeod, L, Medforth, J, Miskimmin, R, Moffat, J, Pickup, M, Prentice, C, Rahman, M, Reda, E, Ross, C, Ryalls, A, Schmid, D, Shergill, N, Snaddon, A, Snell, H, Stevens, L, Waterman, A, Watts, V, Jayne, K, Keirnan, E, Newman, P, Ritchie, G, Rosenfeld, A, Beller, E, Gebski, V, Pillai, A, Anderson, C, Blakesmith, S, Chan, S-Y, Czyniewski, S, Dobbie, A, Doshi, S, Dupuy, A, Eckermann, S, Edwards, M, Fields, N, Flood, K, Ford, S, French, C, Gillies, S, Greig, C, Groshens, M, Gu, J, Guo, Y, Hague, W, Healy, S, Hones, L, Hossain, Z, Howlett, M, Lee, J, Li, L-P, Matthews, T, Micallef, J, Martin, A, Minns, I, Nguyen, A, Papuni, F, Patel, A, Pike, R, Pena, M, Pinto, K, Schipp, D, Schroeder, J, Sim, B, Sodhi, C, Sourjina, T, Sutton, C, Taylor, R, Vlagsma, P, Walder, S, Walker, R, Wong, W, Zhang, J, Zhong, B, Kokkonen, A, Narva, P, Niemi, E-L, Syrjanen, A-M, Lintott, C, Tirimacco, R, Kajosaari, M, Raman, L, Sundvall, J, Tukianen, M, Crimet, D, Sirugue, I, Aubonnet, P

المصدر: Cardiovascular Diabetology

مصطلحات موضوعية: Male, Cardiac & Cardiovascular Systems, Endocrinology, Diabetes and Metabolism, Atorvastatin, Coronary Disease, Fibrate, SECONDARY PREVENTION, ATORVASTATIN, law.invention, Placebos, Randomized controlled trial, Fenofibrate, law, Myocardial infarction, 1102 Cardiorespiratory Medicine and Haematology, Finland, Original Investigation, Hypolipidemic Agents, PLASMA, CHOLESTEROL, Middle Aged, INSULIN, CARDIOVASCULAR-DISEASE, Cardiovascular Diseases, Female, Cardiology and Cardiovascular Medicine, Life Sciences & Biomedicine, medicine.drug, medicine.medical_specialty, medicine.drug_class, Diabetes Complications, Endocrinology & Metabolism, Double-Blind Method, Internal medicine, Diabetes mellitus, Intervention (counseling), medicine, Humans, CORONARY-HEART-DISEASE, Obesity, Triglycerides, Aged, Apolipoproteins B, Bezafibrate, Science & Technology, business.industry, Cholesterol, HDL, Australia, Cholesterol, LDL, medicine.disease, BEZAFIBRATE, FIELD Study Investigators, MYOCARDIAL-INFARCTION, Diabetes Mellitus, Type 2, Cardiovascular System & Hematology, Cardiovascular System & Cardiology, business, HIGH-DENSITY-LIPOPROTEIN, New Zealand

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ecc133c06b82ebac5b1b6558499492fb
http://hdl.handle.net/10044/1/83477

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