Good-quality research in rare diseases: trials and tribulations

Author:

Bolignano Davide,Pisano Anna

Publisher

Springer Science and Business Media LLC

Subject

Nephrology,Pediatrics, Perinatology and Child Health

Reference22 articles.

1. (1984 as amended) Orphan Drug Act, Public Law, No. 97-414, 96 Statute 2049. Available at https://www.gpo.gov/fdsys/pkg/STATUTE-96/pdf/STATUTE-96-Pg2049.pdf

2. (2013) National Center for Advancing Translational Sciences. Office of Rare Disease Research. Frequently asked questions. https://rarediseases.info.nih.gov/about-gard/pages/31/frequently-asked-questions . Accessed 26 October 2015

3. (2005) EURORDIS: European Organisation for Rare Diseases. Rare disease: understanding this public health priority. http://www.eurordis.org/IMG/pdf/princeps_document-EN.pdf . Accessed 26 October 2015

4. Research, N.I.o.H.O.o.R.D. http://rarediseases.info.nih.gov . Accessed 26 October 2015

5. Soliman NA (2012) Orphan kidney diseases. Nephron Clin Pract 120:c194–c199

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