Individual patient (n=1) "trials" in duchenne dystrophy

Neuromuscular Disorders

Volumn 21, Issue 7, 2011, Pages 525-526

  Catlin, Nick ^a   Bettelheim, Karl ^a   Henderson, Iain ^a  

^a Action Duchenne   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE;

DRUG EFFICACY; DRUG RESEARCH; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; GENETIC POLYMORPHISM; GENETIC VARIABILITY; GENOMIC INSTABILITY; HUMAN; LETTER; PERSONALIZED MEDICINE; PRIORITY JOURNAL;

HUMANS; INDIVIDUALIZED MEDICINE; MUSCULAR DYSTROPHY, DUCHENNE; THERAPEUTIC MISCONCEPTION;

EID: 79960082369     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2011.05.003     Document Type: Letter

References (7)

1. Aartsma-Rus A., Advocacy Grp.Individuals The risks of therapeutic misconception and individual patient (n=1) " trials" in rare diseases such as Duchenne dystrophy. Neuromuscular Disorders 2011, 21(1):13-15.
2. Duff G. Back to the future - And to the drawing board. Lancet 2011, 377:371-372.
3. Richard L., Kravitz, et al. Marketing therapeutic precision: potential facilitators and barriers to adoption of n-of-1 trials. Contemporary Clinical Trials 2009, 30:436-445.
4. Richard L., Kravitz, et al. N-of-1 trials of expensive biological therapies a third way?. Arch Intern Med 2008, 168(10):1030-1033.
5. Andrew L., Avinsa, et al. Use of an embedded N-of-1 trial to improve adherence and increase information from a clinical study. Contemporary Clinical Trials 2005, 26(2005):397-401.
6. North Star Database. Available from: http://www.ucl.ac.uk.
7. DMD Registry. Available from: http://www.dmdregistry.org.