Zobrazeno 1 - 6
of 6
pro vyhledávání: '"D. Pangonis"'
Autor:
T. Fields, M. Patterson, T. Bremova-Ertl, G. Belcher, I. Billington, G. C. Churchill, W. Davis, W. Evans, S. Flint, A. Galione, U. Granzer, J. Greenfield, R. Karl, R. Kay, D. Lewi, T. Mathieson, T. Meyer, D. Pangonis, F. M. Platt, L. Tsang, C. Verburg, M. Factor, M. Strupp
Publikováno v:
Trials, Vol 22, Iss 1, Pp 1-15 (2021)
Abstract Background The lack of approved treatments for the majority of rare diseases is reflective of the unique challenges of orphan drug development. Novel methodologies, including new functionally relevant endpoints, are needed to render the deve
Externí odkaz:
https://doaj.org/article/7e79df41cea1412ebe6936c900bbe966
Autor:
Fields, T., M. Patterson, T. Bremova-Ertl, G. Belcher, I. Billington, G. C. Churchill, W. Davis, W. Evans, S. Flint, A. Galione, U. Granzer, J. Greenfield, R. Karl, R. Kay, D. Lewi, T. Mathieson, T. Meyer, D. Pangonis, F. M. Platt, L. Tsang, C. Verburg, M. Factor, M. Strupp
Additional file 1: Supplementary Material I. Video Acquisition, Submission, and Review. Supplementary Material II. Safety Parameters. Supplementary Material III. Data Collection.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::287decb55dffd8a49db1d7db7a9215e2
Autor:
Fields, T., M. Patterson, T. Bremova-Ertl, G. Belcher, I. Billington, G. C. Churchill, W. Davis, W. Evans, S. Flint, A. Galione, U. Granzer, J. Greenfield, R. Karl, R. Kay, D. Lewi, T. Mathieson, T. Meyer, D. Pangonis, F. M. Platt, L. Tsang, C. Verburg, M. Factor, M. Strupp
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___::d258dc821ac636ec7ea33c69a8e069bb
Autor:
Frances M. Platt, William Evans, R. Karl, Tatiana Bremova-Ertl, G. Belcher, S. Flint, T. Fields, L. Tsang, T. Meyer, Marc C. Patterson, D. Lewi, T. Mathieson, J. Greenfield, C. Verburg, Antony Galione, R. Kay, Grant C. Churchill, M. Factor, Michael Strupp, I. Billington, D. Pangonis, W. Davis, U. Granzer
Publikováno v:
Trials
Fields, T; Patterson, M; Bremova-Ertl, T; Belcher, G; Billington, I; Churchill, G C; Davis, W; Evans, W; Flint, S; Galione, A; Granzer, U; Greenfield, J; Karl, R; Kay, R; Lewi, D; Mathieson, T; Meyer, T; Pangonis, D; Platt, F M; Tsang, L; ... (2021). A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. Trials, 22(1), p. 84. BioMed Central 10.1186/s13063-020-05009-3
Trials, Vol 22, Iss 1, Pp 1-15 (2021)
Fields, T; Patterson, M; Bremova-Ertl, T; Belcher, G; Billington, I; Churchill, G C; Davis, W; Evans, W; Flint, S; Galione, A; Granzer, U; Greenfield, J; Karl, R; Kay, R; Lewi, D; Mathieson, T; Meyer, T; Pangonis, D; Platt, F M; Tsang, L; ... (2021). A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia. Trials, 22(1), p. 84. BioMed Central 10.1186/s13063-020-05009-3
Trials, Vol 22, Iss 1, Pp 1-15 (2021)
BackgroundThe lack of approved treatments for the majority of rare diseases is reflective of the unique challenges of orphan drug development. Novel methodologies, including new functionally relevant endpoints, are needed to render the development pr
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1848d38e4af7fcfdc6d591639701540a
http://europepmc.org/articles/PMC7821839
http://europepmc.org/articles/PMC7821839
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