The risks of therapeutic misconception and individual patient (n=1) “trials” in rare diseases such as Duchenne dystrophy-Reference-Cited by-同舟云学术

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

Published:2011-01 Issue:1 Volume:21 Page:13-15
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Aartsma-Rus Annemieke,Furlong Pat,Vroom Elizabeth,van Ommen Gert-Jan,Niks Erik,Straathof Chiara,Verschuuren Jan,Aartsma-Rus Annemieke,Ferlini Alessandra,Hagger Lynn,Heslop Emma,Karcagi Veronika,Kirschner Jan,McCormack Pauline,Moeschen Pat,Muntoni Francesco,Ouillade Marie-Christine,Rahbeck Jes,Rehmann-Sutter Christoph,Rouault Francoise,Sejersen Tomas,Vroom Elizabeth,Woods Simon

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference6 articles.

1. Antisense-mediated modulation of splicing: therapeutic implications for Duchenne muscular dystrophy;Aartsma-Rus;RNA Biol,2010

2. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations;Aartsma-Rus;Hum Mutat,2009

3. Placebos and placebo effects in medicine: a historical overview;Craen;J R Soc Med,1999

4. N-of-1-trial of expensive biological therapies: a third way?;Kravitz;Arch Intern Med,2008

5. Therapeutic efforts in Duchenne muscular dystrophy; the need for a common language between basic scientists and clinicians;Dubowitz;Neuromuscul Disord,2004

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