AADC Gene Therapy for Advanced Parkinson's Disease: Interim Results of a Phase 1b Trial

被引:0
|
作者
Ravina, Bernard [1 ]
Christine, Chadwick W. [2 ]
Larson, Paul S. [2 ]
Van Laar, Amber [3 ]
Richardson, R. Mark [3 ]
Kells, Adrian [1 ]
Boot, Brendon [1 ]
Martin, Alastair [2 ]
Thompson, Marin [2 ]
Bankiewicz, Krystof S. [2 ]
机构
[1] Voyager Therapeut, Cambridge, MA USA
[2] Univ Calif San Francisco, San Francisco, CA 94143 USA
[3] Univ Pittsburgh, Med Ctr, Pittsburgh, PA USA
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
63
引用
收藏
页码:32 / 32
页数:1
相关论文
共 50 条
  • [31] Gene therapy for Parkinson disease: implications from a clinical study of AADC deficiency
    Muramatsu, S.
    HUMAN GENE THERAPY, 2018, 29 (12) : A19 - A20
  • [32] AADC gene therapy for Parkinson disease: Four years of follow-up
    Muramatsu, Shin-ichi
    Fujimoto, Ken-ichi
    Kato, Seiya
    Asari, Sayaka
    Mizukami, Hiroaki
    Ikeguchi, Kunihiko
    Kawakami, Tadataka
    Urabe, Masashi
    Kume, Akihiro
    Sato, Toshihiko
    Watanabe, Eijyu
    Ozawa, Keiya
    Nakano, Imaharu
    NEUROSCIENCE RESEARCH, 2011, 71 : E101 - E101
  • [33] Preliminary results of an open-label, dose-escalation, safety study of AADC gene transfer therapy for Parkinson's disease
    Aminoff, M. J.
    Christine, C. W.
    Bankiewicz, K.
    Starr, P. A.
    Larson, P.
    Mah, R.
    Eberling, J. L.
    Jagust, W. J.
    MOVEMENT DISORDERS, 2007, 22 : S126 - S126
  • [34] Editors' Note: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial
    Galetta, Steven
    Ganesh, Aravind
    Lewis, Ariane
    Siegler, James E., III
    NEUROLOGY, 2022, 99 (06) : 258 - 258
  • [35] Safety and tolerability of IRL790 in Parkinson’s disease with levodopa-induced dyskinesia—a phase 1b trial
    Per Svenningsson
    Anders Johansson
    Dag Nyholm
    Panagiota Tsitsi
    Fredrik Hansson
    Clas Sonesson
    Joakim Tedroff
    npj Parkinson's Disease, 4
  • [36] Association of an interleukin 1B gene polymorphism (-511) with Parkinson's disease in Finnish patients
    Mattila, KM
    Rinne, JO
    Lehtimäki, T
    Röyttä, M
    Ahonen, JP
    Hurme, M
    JOURNAL OF MEDICAL GENETICS, 2002, 39 (06) : 400 - 402
  • [37] Author Response: Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial
    Christine, Chadwick W.
    Richardson, R. Mark
    Van Laar, Amber
    Fine, Elisabeth M.
    Khwaja, Omar S.
    Liang, Grace S.
    Bankiewicz, Krystof
    Larson, Paul S.
    NEUROLOGY, 2022, 99 (06) : 260 - 261
  • [38] Results from a Phase 1b/2 Study of Ibrutinib Combination Therapy in Advanced Urothelial Carcinoma
    Mar, Nataliya
    Zakharia, Yousef
    Falcon, Alejandro
    Morales-Barrera, Rafael
    Mellado, Begona
    Duran, Ignacio
    Oh, Do-Youn
    Williamson, Stephen K.
    Gajate, Pablo
    Arkenau, Hendrik-Tobias
    Jones, Robert J.
    Teo, Min Yuen
    Turan, Tolga
    McLaughlin, Robert T.
    Peltier, Hillary M.
    Chong, Elizabeth
    Atluri, Harisha
    Dean, James P.
    Castellano, Daniel
    CANCERS, 2023, 15 (11)
  • [39] ProSavin® a Dopamine Gene Therapy Approach for Parkinson's Disease: Phase I Clinical Trial
    Palfi, S.
    Gurruchaga, J. -M.
    Ralph, G. S.
    Watts, C.
    Buttery, P.
    Scorer, S.
    Lepetit, H.
    Miskin, J.
    Iwamuro, H.
    Lavisse, S.
    Kas, A.
    Ramelli, A. L.
    Tani, N.
    Dolphin, P.
    Fenelon, G.
    Brugiere, P.
    Kingsman, S.
    Naylor, S.
    Hantraye, P.
    Remy, P.
    Barker, R.
    Mitrophanous, K.
    CELL TRANSPLANTATION, 2013, 22 (05) : 911 - 911
  • [40] ProSavin® a Gene Therapy Approach for Parkinson's Disease: Phase I Clinical Trial Update
    Palfi, Stephane
    Watts, Colin
    Gurrachaga, Jean-Marc
    Buttery, Philip
    Ralph, Scott
    Deeley, Saray
    Lepetit, Helene
    Miskin, James
    Iwamuro, Hirokazu
    Lavisse, Sonia
    Kas, Aurelie
    Ramelli, Anne Laure
    Tani, Naoki
    Dolphin, Patrice
    Abhay, K.
    Gabriel, I.
    Fenelon, Gilles
    Brugiere, Pierre
    Naylor, Stuart
    Hantraye, Philippe
    Remy, Philippe
    Barker, Roger
    Mitrophanous, Kyriacos
    MOLECULAR THERAPY, 2012, 20 : S23 - S23