Quantifying physical decline in juvenile neuronal ceroid lipofuscinosis (Batten disease).

  title={Quantifying physical decline in juvenile neuronal ceroid lipofuscinosis (Batten disease).},
  author={Jennifer M Kwon and Hadie Adams and Paul G. Rothberg and Erika F Augustine and Frederick J. Marshall and E A Deblieck and Amy E Vierhile and Christopher A. Beck and Nicole Newhouse and Jennifer Cialone and Erika Levy and Denia Ramirez-Montealegre and Leon S. Dure and Katherine R Rose and Jonathan W. Mink},
  volume={77 20},
OBJECTIVE To use the Unified Batten Disease Rating Scale (UBDRS) to measure the rate of decline in physical and functional capability domains in patients with juvenile neuronal ceroid lipofuscinosis (JNCL) or Batten disease, a neurodegenerative lysosomal storage disorder. We have evaluated the UBDRS in subjects with JNCL since 2002; during that time, the scale has been refined to improve reliability and validity. Now that therapies are being proposed to prevent, slow, or reverse the course of… CONTINUE READING
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