Journal article icon

Journal article

Clinical trial simulations based on genetic stratification and the natural history of a functional outcome measure in Creutzfeldt-Jakob disease

Abstract:

A major challenge for drug development in neurodegenerative diseases is that adequately powered efficacy studies with meaningful end points typically require several hundred participants and long durations. Prion diseases represent the archetype of brain diseases caused by protein misfolding, the most common subtype being sporadic Creutzfeldt-Jakob disease (sCJD), a rapidly progressive dementia. There is no well-established trial method in prion disease.To establish a more powerful and meanin...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Authors


Burnell, M More by this author
Thompson, A More by this author
Expand authors...
Medical Research Council More from this funder
Department of Health (England) More from this funder
University College London/University College Hospitals Biomedical Research Centre More from this funder
Publisher:
American Medical Association Publisher's website
Journal:
JAMA neurology Journal website
Volume:
73
Issue:
4
Pages:
447-455
Publication date:
2016-04-05
DOI:
EISSN:
2168-6157
ISSN:
2168-6149
URN:
uuid:46475060-00ed-459f-a22f-64e3b5f2b8c7
Source identifiers:
606043
Local pid:
pubs:606043
Language:
English

Terms of use


Metrics



If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP