Friedmann, K. (2020). Supportive care needs in rare disease: evaluation of an expanded access program for Niemann Pick Disease Type C. Presented at the Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Berry-Kravis, E., Chin, J., Jaeger, R., Friedmann, K., Hoffmann, A., Wainer, A., Leonczyk, C., Soorya, L., Farhat, N., Porter, F. (2019). Impact of intrathecal 2-hydroxylpropyl-beta-cyclodextrin (HP-B-CD, VTS-270) on cognitive decline in NPC1. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Jaeger R., Friedmann K., Leonczyk C., Hoffmann A., Wainer A., … Porter, F. (2019). Effects of Hydroxypropyl-beta-cyclodextrin HB-B-CD, VTS-270 in children with NPC1 age 5 and under: Comparison to natural history. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Friedmann, K. (2018). Updates to the implementation of a national, multi-site, expanded access program for the treatment of neurological disease in Niemann Pick disease type C1 with intrathecal VTS-270. Michael, Marcia and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research, Tucson, AZ.
Berry-Kravis, E., Seinfeld, S., Chin, J., Friedmann, K., O’Hara, K. (2018). Protriptyline and severe cataplexy in Niemann-Pick type C1. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Berry-Kravis, E., Chin, J., Friedmann, K., Wainer, A., Leonczyk, C., Gonring, K., Hoffmann, A. (2018). Impact of treatment with intrathecal 2-hydroxy-beta-cyclodextrin (HP-B-CD, VTS-270) on cognitive decline in NPC1. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Berry-Kravis, E., Chin, J., Friedmann, K., Hernandez, M., Nadelman, A., … O’Keefe J. (2018). Treatment of very young children with intrathecal 2-hydroxy-beta-cyclodextrin (VTS-270): Experience with 6 NPC1 patients. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Friedmann, K. (2017). Implementation of a national, multisite, expanded access program for Niemann Pick disease type C1. Oral presentation, National Niemann Pick Disease Foundation Annual Family Conference, Chicago, IL.
Berry-Kravis, E., O’Keefe, J., Hoffmann, A., LaGorio, L., Friedmann, K., … Ardestani, M. (2017). Arrest of progression of Niemann-Pick type C1 (NPC) with intrathecal 2-hydroxypropyl-ß-cyclodextrin (VTS-270) in multiple neurological domains for 3 years. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Friedmann, K., Hernandez, M., Chin, J., Mishos, C., … Berry-Kravis, E. (2016). Implementation of a national, multi-site, expanded access program for the treatment of neurological disease in Niemann Pick disease type C1, with intrathecal VTS-270. Michael, Marcia and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research, Tucson, AZ.
Berry-Kravis, E., O’Keefe, J., Hoffmann, A., Winston, A., Stoner, R., … Friedmann, K. (2016). Performance on objective clinical efficacy outcome measures during long-term cyclodextrin (VTS-270) treatment in NPC. Michael, Marcia & Christa Parseghian Scientific Conference, Tucson, AZ.
Berry-Kravis, E., O’Keefe, J., Hoffmann, A., Winston, A., Stoner, R., … Friedmann, K. (2016). Performance on objective clinical efficacy outcome measures during long-term cyclodextrin (VTS-270) treatment in NPC. National Niemann-Pick Disease Foundation Family Conference, Boston, MA.
