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Meet the Scientists

Scientific collaborators in the NPC and broader scientific community

  • David Begley, King’s College London: CNS distribution of hydroxypropyl-beta-cyclodextrin (HPBCD)
  • Ashley Bush, University of Melbourne: Measurement of metals in NPC1 mouse tissues
  • Keith Duncan, University of Michigan: HPBCD-induced ototoxicity
  • Larry Goldstein, University of California San Diego: Evaluation of compounds that improve mitochondrial viability
  • Paul Helquist and Olaf Wiest, University of Notre Dame; Fred Maxfield, Cornell Weill; Ed Holson, Broad Institute: Evaluation of HDAC inhibitors for treatment of NPC1 disease
  • Danny Manor, Case Western Reserve University: Evaluation of tocopherol processing
  • Ralph Nixon, Nathan S. Kline Institute: Similarities between NPC and Alzheimer mouse models
  • Bill Pavan, National Human Genome Research Institute: Effect of NAC on NPC1 disease progression and gene therapy in NPC1 disease
  • Fran Platt, University of Oxford; Mario Cortina-Borja, University College London: Analysis of biomarkers
  • Heiko Runz, University of Heidelberg: Effect of TMEM97 inhibition in vivo
  • Lajos Szente, CycloLab: Cyclodextrin biochemistry and mechanism of action
  • Wei Zheng, National Center for Advancing Translational Sciences: Examination of combined tocopherol/cyclodextrin therapy
  • Charles Venditti and Bill Pavan, National Human Genome Research Institute : Gene therapy in NPC1 disease
  • Vytautas Verselis and Helmuth Sanchez, Albert Einstein College of Medicine: Effect of cyclodextrin on hearing and hair cells in an NPC1 mouse model

NPC Research

Interested in learning more about NPC?  Put your science cap on and explore some of the different topics in NPC research!

Collaboration at Work

Therapeutic Advancements

Diagnostic and Biomarker Advancements

Exciting Basic Research

Origins of Cyclodextrin as a Therapy for NPC disease

Tiger and John are NPC1 heterozygote cats.  Upon retirement from status as breeder males, both cats were adopted from the Niemann-Pick C feline colony by a member of the NPC community.