An Open Access Journal
Letter from the Editors
M. Ian Phillips, PhD, DSc, FAHA and Tim Coté, MD, MPH
Physician and Patient Perceptions Regarding Physician Training in Rare Diseases: The Need for Stronger Educational Initiatives for Physicians
Recent FDA Orphan Drug Designations I. Without Cancer Drugs(March 25, 2013, to May 30, 2013)
Toward European Guidelines for PKU: How to Speak a Common Language?
Annemiek van Wegberg, Msc, François Maillot, MD, PhD, and Francjan van Spronsen, MD, PhD
Phenotypic Variability in Individuals with Type V Osteogenesis Imperfecta with Identical IFITM5 Mutations
Jamie Fitzgerald, PhD; Paul Holden, PhD; Hollis Wright, PhD; Beth Wilmot, PhD; Abigail Hata, CGC; Robert D. Steiner, MD; and Don Basel, MD
Cochrane Corner Enzymes for Inborn Errors: Laronidase (Aldurazyme®) for Treating Mucopolysaccharidosis Type I
Elisabeth Jameson, MBBCh, BSc (Hons), MSc, MRCPCH and Simon Jones, MBChB, BSc (Hons), MRCPCH
Copyright 2017 Scientific Communications Group. All Rights Reserved.
Website Designed by Hudson Horizons