Orphanet Journal of Rare Diseases
Articles
- POSITION STATEMENT
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III
Published on: 26 June 2017 - RESEARCH
- RESEARCH
Do investors value the FDA orphan drug designation?
Published on: 19 June 2017 - RESEARCH
Prenatal brain disruption in isolated sulfite oxidase deficiency
Published on: 19 June 2017 - REVIEW
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