Skip to main navigation Skip to search Skip to main content

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

  • Daniel S. Ory
  • , Elizabeth A. Ottinger
  • , Nicole Yanjanin Farhat
  • , Kelly A. King
  • , Xuntian Jiang
  • , Lisa Weissfeld
  • , Elizabeth Berry-Kravis
  • , Cristin D. Davidson
  • , Simona Bianconi
  • , Lee Ann Keener
  • , Ravichandran Rao
  • , Ariane Soldatos
  • , Rohini Sidhu
  • , Kimberly A. Walters
  • , Xin Xu
  • , Audrey Thurm
  • , Beth Solomon
  • , William J. Pavan
  • , Bernardus N. Machielse
  • , Mark Kao
    • Steven A. Silber, John C. McKew, Carmen C. Brewer, Charles H. Vite, Steven U. Walkley, Christopher P. Austin, Forbes D. Porter

Research output: Contribution to journal › Article › peer-review

299   Link opens in a new tab Scopus citations

Fingerprint

Dive into the research topics of 'Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial'. Together they form a unique fingerprint.
Sort by

Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science

Neuroscience

Biochemistry, Genetics and Molecular Biology

Keyphrases