The Medical Research Council prion disease rating scale: a new outcome measure for prion disease therapeutic trials developed and validated using systematic observational studies.

Journal article

Progress in therapeutics for rare disorders like prion disease is impeded by the lack of validated outcome measures and a paucity of natural history data derived from prospective observational studies. The first analysis of the U.K. National Prion Monitoring Cohort involved 1337 scheduled clinical assessments and 479 telephone assessments in 437 participants over 373 patient-years of follow-up. Scale development has included semi-quantitative and qualitative carer interviews, item response mo...

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Authors: Thompson, A; Lowe, J; Fox, Z; Lukic, A; Porter, M

• Publication status: Published
• Journal: Brain : a journal of neurology
• Volume: 136
• Issue: Pt 4
• Pages: 1116-1127
• Publication date: 2013-04-01
• DOI: 10.1093/brain/awt048
• EISSN: 1460-2156
• ISSN: 0006-8950
• Language: English
• Keywords: Prion Diseases; Great Britain; Health Surveys; Creutzfeldt-Jakob Syndrome; Female; Cohort Studies; Adult; Psychiatric Status Rating Scales; Male; Aged, 80 and over; Time Factors; Disease Progression; Clinical Trials as Topic; Aged; Humans; Adolescent; Middle Aged; Young Adult