The risks of therapeutic misconception and individual patient (n=1) "trials" in rare diseases such as Duchenne dystrophy

Neuromuscular Disorders

Volumn 21, Issue 1, 2011, Pages 13-15

  Aartsma Rus, Annemieke ^a   Furlong, Pat ^b   Vroom, Elizabeth ^c   van Ommen, Gert Jan ^a   Niks, Erik ^a   Straathof, Chiara ^a   Verschuuren, Jann ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b President of the Parent Project Muscular Dystrophy   (United States)

^c President of the United Parent Project and of the Dutch Duchenne Parent Project   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; PLACEBO;

CLINICAL TRIAL; DISEASE MODEL; DRUG EFFICACY; DRUG SAFETY; DUCHENNE MUSCULAR DYSTROPHY; EDITORIAL; EXON SKIPPING; GENE MUTATION; HUMAN; LONG TERM CARE; NONHUMAN; PHARMACODYNAMICS; PRIORITY JOURNAL; RISK ASSESSMENT;

CLINICAL TRIALS AS TOPIC; HUMANS; INDIVIDUALIZED MEDICINE; MUSCULAR DYSTROPHY, DUCHENNE; THERAPEUTIC MISCONCEPTION;

EID: 78650303456     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2010.09.012     Document Type: Editorial

References (6)

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2. Aartsma-Rus A., Fokkema I., Verschuuren J., et al. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat 2009, 30:293-299.
3. Craen A.J., Kaptchuk T.J., Tijssen J.G., et al. Placebos and placebo effects in medicine: a historical overview. J R Soc Med 1999, 92:511-515.
4. Kravitz R.L., Duan N., White R.H. N-of-1-trial of expensive biological therapies: a third way?. Arch Intern Med 2008, 168:1030-1033.
5. Dubowitz V. Therapeutic efforts in Duchenne muscular dystrophy; the need for a common language between basic scientists and clinicians. Neuromuscul Disord 2004, 14:451-455.
6. Muntoni F. The development of antisense oligonucleotide therapies for Duchenne muscular dystrophy: report on a TREAT-NMD workshop hosted by the European Medicines Agency (EMA), on September 25th 2009. Neuromuscul Disord 2010, 20:355-362.