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.
A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustatRemenova T, Morand O, Amato D, Chadha-Boreham H, Tsurutani S, Marquardt T
Orphanet Journal of Rare Diseases 2015, 10 :81 (19 June 2015)
Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countriesBosch AM, Burlina A, Cunningham A, Bettiol E, Moreau-Stucker F, Benmedjahed K, Regnault A
Orphanet Journal of Rare Diseases 2015, 10 :80 (18 June 2015)
Ferdinandusse S, Friederich M, Burlina A, Ruiter J, Coughlin C, Dishop M, Gallagher R, Bedoyan J, Vaz F, Waterham H, Gowan K, Chatfield K, Bloom K, Bennett M, Elpeleg O, Van Hove J, Wanders R
Orphanet Journal of Rare Diseases 2015, 10 :79 (18 June 2015)