The following new articles have just been published in Orphanet Journal of Rare Diseases
For articles using Author Version-first publication you will see a provisional PDF corresponding to the accepted manuscript. In these instances, the fully formatted Final Version PDF and full text (HTML) versions will follow in due course.
Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in GermanySchreiber-Katz O, Klug C, Thiele S, Schorling E, Zowe J, Reilich P, Nagels KH, Walter MC
Orphanet Journal of Rare Diseases 2014, 9 :210 (18 December 2014)
An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1AAttarian S, Vallat J, Magy L, Funalot B, Gonnaud P, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre M, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D
Orphanet Journal of Rare Diseases 2014, 9 :199 (18 December 2014)