CLN3 Research Tools and Resources
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Rare Disease and Orphan Drugs
Beachy, S.H., Johnson, S.G., Olson, S. and A.C. Berger. (2014) Drug Repurposing and Repositioning – Workshop Summary. Washington D.C.: National Academies Press.
Gliklich, R.E., Dreyer, N.A., Leavy, M.B. (2014) Registries for Evaluating Patient Outcomes: A User’s Guide [Internet]. 3rd edition. Rockville (MD): Agency for Healthcare Research and Quality (US).
Hernberg-Ståhl, E. and M. Reljanović (2013). Orphan Drugs: Understanding the Rare Disease Market and its Dynamics, 1st edition. Cambridge: Woodhead Publishing Limited.
Institute of Medicine. (2010) Rare Diseases and Orphan Products: Accelerating Research and Development 1st Edition, Washington (DC):National Academies Press.
Mole, S. and R.E. Williams. (2013) Neuronal Ceroid-Lipofuscinoses in GeneReviews®. Seattle (WA): University of Washington, Seattle.
Mole, S. Williams, R. and H. Goebel. (2011) The Neuronal Ceroid Lipofusinoses (Batten Disease) (2 ed.) Oxford: Oxford University Press.
Reilly, P.R. (2015) Orphan: The Quest to Save Children with Rare Genetic Disorders. Cold Spring Harbor, New York: Cold Spring Harbor Laboratory Press.
Sireau N. (2013) Rare Diseases: Challenges and Opportunities for Social Entrepreneurs (1st Ed) Austin TX: Greenleaf Publishing.
Designing a plan for drug discovery in rare pediatric neurodegenerative disease
Written by: Danielle Kerkovich, PhD and Amy Drew, MA