Published in
BMJ Publishing Group, Journal of Neurology, Neurosurgery and Psychiatry, 12(86), p. 1291-1298, 2015
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Short-interval observational data to inform clinical trial design in Huntington's disease
Journal article published in 2015 by
Nz Z. Hobbs,
Re E. Farmer,
Em M. Rees,
Jh H. Cole 
,
Salman Haider,
Ib B. Malone,
Reiner Sprengelmeyer,
Hans Johnson,
Hp-P. Mueller,
Sd D. Sussmuth,
Ra A. C. Roos,
Alexandra Durr,
Chris Frost,
Ri I. Scahill,
Bernhard Landwehrmeyer
and other authors.
,
Salman Haider,
Ib B. Malone,
Reiner Sprengelmeyer,
Hans Johnson,
Hp-P. Mueller,
Sd D. Sussmuth,
Ra A. C. Roos,
Alexandra Durr,
Chris Frost,
Ri I. Scahill,
Bernhard Landwehrmeyer
and other authors.
This paper is available in a repository.
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Abstract
To evaluate candidate outcomes for disease-modifying trials in Huntington's disease (HD) over 6-month, 9-month and 15-month intervals, across multiple domains. To present guidelines on rapid efficacy readouts for disease-modifying trials.