- Beyond Batten Disease Foundation - https://beyondbatten.org -




CLN3 Research Tools and Resources

View/Download the PDF [1]

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. [2]

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). [3]

Hernberg-Ståhl, E. and M. Reljanović (2013). Orphan Drugs: Understanding the Rare Disease Market and its Dynamics, 1st edition. Cambridge: Woodhead Publishing Limited.  [4]

Institute of Medicine. (2010) Rare Diseases and Orphan Products: Accelerating Research and Development 1st Edition, Washington (DC):National Academies Press. [5]

Mole, S. and R.E. Williams. (2013) Neuronal Ceroid-Lipofuscinoses in GeneReviews®. Seattle (WA): University of Washington, Seattle.  [6]

Mole, S. Williams, R. and H. Goebel. (2011) The Neuronal Ceroid Lipofusinoses (Batten Disease) (2 ed.) Oxford: Oxford University Press.  [7]

Reilly, P.R. (2015) Orphan: The Quest to Save Children with Rare Genetic Disorders. Cold Spring Harbor, New York: Cold Spring Harbor Laboratory Press.  [8]

Sireau N. (2013) Rare Diseases: Challenges and Opportunities for Social Entrepreneurs (1st Ed) Austin TX: Greenleaf Publishing. [9]


Designing a plan for drug discovery in rare pediatric neurodegenerative disease [10]
Written by: Danielle Kerkovich, PhD and Amy Drew, MA

BBDF strategy [11][/vc_column_text][/vc_column][/vc_row]