ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)


Cleary M., Muschol N., Luz Couce M., Harmatz P., Lee J., Lin S., ...More

15th Annual Research Meeting of the WORLDSymposium(TM), Florida, United States Of America, 4 - 07 February 2019, vol.126 identifier

  • Publication Type: Conference Paper / Summary Text
  • Volume: 126
  • Doi Number: 10.1016/j.ymgme.2018.12.083
  • City: Florida
  • Country: United States Of America