Orphanet Journal of Rare Diseases | Home page

Orphanet Journal of Rare Diseases | Home page





Orphanet Journal of Rare Diseases

Articles

• Recent
• Most accessed

Recent articles RSS

1. 
   
2. POSITION STATEMENT

   Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III

   Arunabha Ghosh, Elsa Shapiro, Stewart Rust, Kathleen Delaney, Samantha Parker, Adam J Shaywitz, Adelaida Morte, Gillian Bubb, Maureen Cleary, Tien Bo, Christine Lavery, Brian W Bigger and Simon A Jones

   Published on: 26 June 2017
3. RESEARCH

   Maternal use of selective serotonin reuptake inhibitors during pregnancy is associated with Hirschsprung’s disease in newborns – a nationwide cohort study

   Sebastian Werngreen Nielsen, Perniller Møller Ljungdalh, Jan Nielsen, Bente Mertz Nørgård and Niels Qvist

   Published on: 20 June 2017
4. RESEARCH

   Do investors value the FDA orphan drug designation?

   Kathleen L. Miller

   Published on: 19 June 2017
5. RESEARCH

   Prenatal brain disruption in isolated sulfite oxidase deficiency

   Hsiu-Fen Lee, Ching-Shiang Chi, Chi-Ren Tsai, Hung-Chieh Chen and I-Chun Lee

   Published on: 19 June 2017
6. REVIEW

   Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders

   Kyeongsoon Kim, Hynda K. Kleinman, Hahn-Jun Lee and Kalipada Pahan

   Published on: 17 June 2017

View all articles