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BioMarin Commitment

The BioMarin Commitment

BioMarin is a world leader in developing innovative treatments for rare diseases

NCLs, collectively known as Batten disease, are the leading cause of dementia in children and adolescents. They are characterized by severe and progressive neurodegeneration and debilitating symptoms. Unfortunately, management resources are much needed.1,2

CLN2 disease, one of the most common forms of NCL,2,3 has been a focus of research and drug development at BioMarin for the last decade. As such, BioMarin has been committed to the CLN2 disease community by:

  • Providing resources and educational materials for healthcare professionals and families
  • Promoting advocacy groups and the value of connecting with the community

References: 1. Schulz A, Kohlschütter A, Mink J, Simonati A, Williams R. NCL diseases – clinical perspectives. Biochimica et Biophysica Acta. 2013;1832:1801-1806. 2. Mole SE, Williams RE. Neuronal ceroid-lipofuscinoses. 2001 Oct 10 [Updated 2013 Aug 1]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [internet]. Seattle, WA: University of Washington; 1993-2016. 3. Haltia M. The neuronal ceroid-lipofuscinoses: from past to present. Biochimica et Biophysica Acta. 2006;1762:850-856.