• Sanfilippo syndrome : consensus guidelines for clinical care 

      Muschol, Nicole; Giugliani, Roberto; Jones, Simon A.; Muenzer, Joseph; Smith, Nicholas J. C.; Whitley, Chester B.; Donnell, Megan; Drake, Elise; Elvidge, Kristina; Melton, Lisa; O’Neill, Cara (2022) [Artigo de periódico]
      Sanfilippo syndrome is a group of rare, complex, and progressive neurodegenerative lysosomal storage disorders that is characterized by childhood dementia. The clinical management of patients with progressive neurological ...