Beyond Batten Disease Foundation

Join us in our mission to find a cure for Batten disease and prevent hundreds of similar rare genetic diseases that claim the lives of thousands of children every year.

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      Beyond Batten Disease Foundation:

      3305 Steck Ave., Suite 200
      Austin, TX 78757
      Phone: 512.275.2600
      info@beyondbatten.org

      Submit an Inquiry »

  • Understanding Batten
    • About Batten Disease

      • What is Batten?
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      Beyond Batten Disease Foundation:

      3305 Steck Ave., Suite 200
      Austin, TX 78757
      Phone: 512.275.2600
      info@beyondbatten.org

      Submit an Inquiry »

  • Research
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      Beyond Batten Disease Foundation
      Phone: 512.275.2600
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Resources

Beyond Batten Disease Foundation  ⁄  Research  ⁄  Resources

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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.

Publications

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

BBDF strategy

What We Know

  • State of the Science
  • CLN3 Gene
  • Screening

Foundation Driven

  • Our Strategy
  • Research Activities
  • Batten-1

Contact Us

  • 512.275.2600
  • info@beyondbatten.org
  • 3305 Steck Ave., Suite 200, Austin Texas, 78757

Connect With Us

What’s New?

  • My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility March 2, 2023
  • Rare Disease Day 2023 – A Discussion February 23, 2023
  •  THERANEXUS, BBDF AND CARDIFF UNIVERSITY PRESENT THEIR NOVEL RESEARCH ON BATTEN-1 AND BATTEN DISEASE AT WORLDSYMPOSIUM 2023  February 22, 2023

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