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

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

Additional file 1 of 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

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

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

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)

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

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Health care assistants and health visiting

Autor: I, Billington, R, Rudkin, R, Salmon

Publikováno v: Health visitor. 65(9)

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::92431efb459cb72dcc3a1179b8aa043a
https://pubmed.ncbi.nlm.nih.gov/1468958