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pro vyhledávání: '"Platt F"'
Akademický článek
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Autor:
Fields, T, Bremova, T M, Billington, I, Churchill, G C, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M
Publikováno v:
Fields, T; Bremova, T M; Billington, I; Churchill, G C; Evans, W; Fields, C; Galione, A; Kay, R; Mathieson, T; Martakis, K; Patterson, M; Platt, F; Factor, M; Strupp, M (2023). N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study. Trials, 24(1), p. 361. BioMed Central 10.1186/s13063-023-07399-6
Background Niemann-Pick disease type C (NPC) is a rare autosomal recessive neurodegenerative lysosomal disease characterized by multiple symptoms such as progressive cerebellar ataxia and cognitive decline. The modified amino acid N-acetyl-leucine ha
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c70744407af9d5c6c4fd3a57351fa078
https://boris.unibe.ch/183014/1/s13063-023-07399-6.pdf
https://boris.unibe.ch/183014/1/s13063-023-07399-6.pdf
Autor:
Fields, T, M. Bremova, T, Billington, I, Churchill, GC, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M
Additional file 3. SPIRIT Checklist for Trials.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7c9dd5c3a5e57c0bbfeb55b866e4a33c
Autor:
Fields, T, M. Bremova, T, Billington, I, Churchill, GC, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M
Additional file 2: Supplementary Table 1. Parent Study schedule of enrolment, interventions, and assessments. Supplementary Table 2. Extension Phase schedule of enrolment, interventions, and assessments.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2a48036a62bde51d5e3e15c9edfad71e
Autor:
Fields, T, M. Bremova, T, Billington, I, Churchill, GC, Evans, W, Fields, C, Galione, A, Kay, R, Mathieson, T, Martakis, K, Patterson, M, Platt, F, Factor, M, Strupp, M
Additional file 1: Supplementary Material I. Safety Parameters. Supplementary Material II. Data Collection.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::abd63cbd590725326ccacf66123bbffd
Autor:
D’Amore, S., Sano, H., Chappell, D., Chiarugi, D., Baker, O., Page, K., Ramaswami, U., Johannesdottir, F., Cox, T., Deegan, P., Poole, K., Platt, F., Banka, S., Chakrapani, A., Geberhiwot, T., Hughes, D., Jones, S., Lachmann, R., Santra, S., Sharma, R., Vellodi, A.
Publikováno v:
Radiology
Background Patients with Gaucher disease (GD) have a high risk of fragility fractures. Routine evaluation of bone involvement in these patients includes radiography and repeated dual-energy x-ray absorptiometry (DXA). However, osteonecrosis and bone
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fdd777858c1163a09ffcd3ba9fca2795
https://europepmc.org/articles/PMC7614382/
https://europepmc.org/articles/PMC7614382/
Publikováno v:
Journal of Medicinal Chemistry. 65:5867-5869
Akademický článek
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Autor:
D’Amore, Simona, Page, Kathleen, Donald, Aimée, Taiyari, Khadijeh, Tom, Brian, Deegan, Patrick, Tan, Chong Y., Poole, Kenneth, Jones, Simon A., Mehta, Atul, Hughes, Derralynn, Sharma, Reena, Lachmann, Robin H., Chakrapani, Anupam, Geberhiwot, Tarekegn, Santra, Saikat, Banka, Siddarth, Cox, Timothy M., Cox, T. M., Platt, F. M., Banka, S., Chakrapani, A., Deegan, P. B., Geberhiwot, T., Hughes, D. A., Jones, S., Lachmann, R. H., Santra, S., Sharma, R., Vellodi, A.
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-22 (2021)
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases
Background The Gaucher Investigative Therapy Evaluation is a national clinical cohort of 250 patients aged 5–87 years with Gaucher disease in the United Kingdom—an ultra-rare genetic disorder. To inform clinical decision-making and improve pathop
Autor:
Gautron, A.S., Giquel, B., Beaudoin, L., Autrusseau, E., Speak, A., Platt, F., Kemp, S., Pujol, A., Aubourg, P., Lehuen, A.
Publikováno v:
In Journal of Neuroimmunology 2010 229(1):204-211