共 50 条
- [1] Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC studyMED, 2024, 5 (09):Percy, Alan K.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USANeul, Jeffrey L.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Dept Pediat, Vanderbilt Kennedy Ctr, Nashville, TN 37232 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USABenke, Timothy A.论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Sch Med, Dept Pediat, Childrens Hosp Colorado, Aurora, CO 80045 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USABerry-Kravis, Elizabeth M.论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Dept Pediat Neurol Sci & Biochem, Med Ctr, Chicago, IL 60612 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USAGlaze, Daniel G.论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Baylor Coll Med, Dept Pediat & Neurol, Houston, TX 77030 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USAMarsh, Eric D.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Childrens Hosp Philadelphia, Perelman Sch Med, Dept Neurol & Pediat,Div Child Neurol, Philadelphia, PA 19104 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USAAn, Di论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USABishop, Kathie M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USAYouakim, James M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL 35233 USA
- [2] Trofinetide for the treatment of Rett syndrome Long-term safety and efficacy results of the 32-month, open-label LILAC-2 studyMED, 2024, 5 (10):Percy, Alan K.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USANeul, Jeffrey L.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Vanderbilt Kennedy Ctr, Dept Pediat, Nashville, TN USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USABenke, Timothy A.论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Sch Med, Childrens Hosp Colorado, Dept Pediat, Aurora, CO USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USABerry-Kravis, Elizabeth M.论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Med Ctr, Dept Pediat, Chicago, IL USA Rush Univ, Med Ctr, Dept Neurol Sci, Chicago, IL USA Rush Univ, Med Ctr, Dept Biochem, Chicago, IL USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USAGlaze, Daniel G.论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Baylor Coll Med, Dept Pediat & Neurol, Houston, TX USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USAMarsh, Eric D.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Childrens Hosp Philadelphia, Dept Neurol, Div Child Neurol,Perelman Sch Med, Philadelphia, PA USA Univ Penn, Perelman Sch Med, Dept Pediat, Div Child Neurol,Childrens Hosp Philadelphia, Philadelphia, PA USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USABarrett, Amy M.论文数: 0 引用数: 0 h-index: 0机构: RTI Hlth Solut, Patient Ctr Outcomes Assessment, Res Triangle Pk, NC USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USAAn, Di论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USABishop, Kathie M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USAYouakim, James M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Univ Alabama Birmingham, Dept Pediat, Birmingham, AL USA
- [3] Trofinetide for the treatment of Rett syndrome: an open-label study in girls 2 to 4 years of age ()NEUROLOGY, 2023, 100 (17)论文数: 引用数: h-index:机构:Ryther, Robin论文数: 0 引用数: 0 h-index: 0机构: Washington Univ, Sch Med, St Louis, MO USA Univ Alabama Birmingham, Birmingham, AL USAMarsh, Eric论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA USA Univ Alabama Birmingham, Birmingham, AL USAFeyma, Timothy论文数: 0 引用数: 0 h-index: 0机构: Gillette Childrens Specialty Healthcare, St Paul, MN USA Univ Alabama Birmingham, Birmingham, AL USALieberman, David论文数: 0 引用数: 0 h-index: 0机构: Boston Childrens Hosp, Boston, MA USA Univ Alabama Birmingham, Birmingham, AL USA论文数: 引用数: h-index:机构:Benke, Timothy论文数: 0 引用数: 0 h-index: 0机构: Univ Colorado, Childrens Hosp Colorado, Sch Med, Boulder, CO USA Univ Alabama Birmingham, Birmingham, AL USAGlaze, Daniel论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Baylor Coll Med, Houston, TX USA Univ Alabama Birmingham, Birmingham, AL USABerry-Kravis, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Childrens Hosp, Chicago, IL USA Univ Alabama Birmingham, Birmingham, AL USAAnanth, Amitha论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Birmingham, AL USA Univ Alabama Birmingham, Birmingham, AL USABuhrfiend, Colleen论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Med Ctr, Chicago, IL USA Univ Alabama Birmingham, Birmingham, AL USAStankovic, Srdjan论文数: 0 引用数: 0 h-index: 0机构: Acad Pharmaceut Inc, San Diego, CA USA Univ Alabama Birmingham, Birmingham, AL USABishop, Kathie论文数: 0 引用数: 0 h-index: 0机构: Acad Pharmaceut Inc, San Diego, CA USA Univ Alabama Birmingham, Birmingham, AL USADarwish, Mona论文数: 0 引用数: 0 h-index: 0机构: Acad Pharmaceut Inc, San Diego, CA USA Univ Alabama Birmingham, Birmingham, AL USAYouakim, James论文数: 0 引用数: 0 h-index: 0机构: Acad Pharmaceut Inc, San Diego, CA USA Univ Alabama Birmingham, Birmingham, AL USA
- [4] An open-label study of trofinetide for the treatment of Rett syndrome in girls 2 to 4 years of ageANNALS OF NEUROLOGY, 2022, 92 : S191 - S192Percy, A.论文数: 0 引用数: 0 h-index: 0Ryther, R.论文数: 0 引用数: 0 h-index: 0Marsh, E.论文数: 0 引用数: 0 h-index: 0Feyma, T.论文数: 0 引用数: 0 h-index: 0Lieberman, D.论文数: 0 引用数: 0 h-index: 0Neul, J.论文数: 0 引用数: 0 h-index: 0Benke, T.论文数: 0 引用数: 0 h-index: 0Glaze, D.论文数: 0 引用数: 0 h-index: 0Berry-Kravis, E.论文数: 0 引用数: 0 h-index: 0Ananth, A.论文数: 0 引用数: 0 h-index: 0Buhrfiend, C.论文数: 0 引用数: 0 h-index: 0Stankovic, S.论文数: 0 引用数: 0 h-index: 0Bishop, K.论文数: 0 引用数: 0 h-index: 0Darwish, M.论文数: 0 引用数: 0 h-index: 0Youakim, J.论文数: 0 引用数: 0 h-index: 0Arkilo, D.论文数: 0 引用数: 0 h-index: 0
- [5] Trofinetide: a pioneering treatment for Rett syndromeTRENDS IN PHARMACOLOGICAL SCIENCES, 2023, 44 (10) : 740 - 741Parent, Harrison论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Sch Med, Dept Pharmacol, Nashville, TN 37235 USA Vanderbilt Univ, Warren Ctr Neurosci Drug Discovery, Nashville, TN 37235 USA Vanderbilt Univ, Sch Med, Dept Pharmacol, Nashville, TN 37235 USAFerranti, Anthony论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Sch Med, Dept Pharmacol, Nashville, TN 37235 USA Vanderbilt Univ, Warren Ctr Neurosci Drug Discovery, Nashville, TN 37235 USA Vanderbilt Univ, Sch Med, Dept Pharmacol, Nashville, TN 37235 USA论文数: 引用数: h-index:机构:
- [6] Trofinetide for the treatment of Rett syndrome: a randomized phase 3 studyNATURE MEDICINE, 2023, 29 (06) : 1468 - +Neul, Jeffrey L.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USAPercy, Alan K.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Birmingham, AL USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USABenke, Timothy A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Colorado, Aurora, CO USA Univ Colorado, Sch Med, Aurora, CO USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USABerry-Kravis, Elizabeth M.论文数: 0 引用数: 0 h-index: 0机构: Rush Univ, Med Ctr, Chicago, IL USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USAGlaze, Daniel G.论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Houston, TX USA Baylor Coll Med, Houston, TX USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USAMarsh, Eric D.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Philadelphia, PA USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USALin, Tim论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USAStankovic, Serge论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USABishop, Kathie M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USAYouakim, James M.论文数: 0 引用数: 0 h-index: 0机构: Acadia Pharmaceut Inc, San Diego, CA 92130 USA Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USA
- [7] Development of trofinetide for the treatment of Rett syndrome: from bench to bedsideFRONTIERS IN PHARMACOLOGY, 2024, 14Kennedy, Melissa论文数: 0 引用数: 0 h-index: 0机构: Int Rett Syndrome Fdn, Cincinnati, OH USA Int Rett Syndrome Fdn, Cincinnati, OH USAGlass, Larry论文数: 0 引用数: 0 h-index: 0机构: Neuren Pharmaceut Ltd, Melbourne, Vic, Australia Int Rett Syndrome Fdn, Cincinnati, OH USAGlaze, Daniel G.论文数: 0 引用数: 0 h-index: 0机构: Texas Childrens Hosp, Baylor Coll Med, Houston, TX USA Int Rett Syndrome Fdn, Cincinnati, OH USAKaminsky, Steve论文数: 0 引用数: 0 h-index: 0机构: Int Rett Syndrome Fdn, Cincinnati, OH USA Int Rett Syndrome Fdn, Cincinnati, OH USAPercy, Alan K.论文数: 0 引用数: 0 h-index: 0机构: Univ Alabama Birmingham, Div Pediat Neurol, Birmingham, AL USA Int Rett Syndrome Fdn, Cincinnati, OH USANeul, Jeffrey L.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Vanderbilt Kennedy Ctr, Med Ctr, Nashville, TN USA Int Rett Syndrome Fdn, Cincinnati, OH USAJones, Nancy E.论文数: 0 引用数: 0 h-index: 0机构: Neuren Pharmaceut Ltd, Melbourne, Vic, Australia Int Rett Syndrome Fdn, Cincinnati, OH USATropea, Daniela论文数: 0 引用数: 0 h-index: 0机构: Trinity Coll Dublin, Inst Neurosci, Dublin, Ireland Int Rett Syndrome Fdn, Cincinnati, OH USAHorrigan, Joseph P.论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Dept Psychiat & Behav Sci, Durham, NC USA Int Rett Syndrome Fdn, Cincinnati, OH USANues, Paige论文数: 0 引用数: 0 h-index: 0机构: Int Rett Syndrome Fdn, Cincinnati, OH USA Int Rett Syndrome Fdn, Cincinnati, OH USABishop, Kathie M.论文数: 0 引用数: 0 h-index: 0机构: ACADIA Pharmaceut Inc, San Diego, CA 92130 USA Int Rett Syndrome Fdn, Cincinnati, OH USAYouakim, James M.论文数: 0 引用数: 0 h-index: 0机构: ACADIA Pharmaceut Inc, San Diego, CA 92130 USA Int Rett Syndrome Fdn, Cincinnati, OH USA
- [8] Randomized open-label trial of dextromethorphan in Rett syndromeNEUROLOGY, 2017, 89 (16) : 1684 - 1690Smith-Hicks, Constance L.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAGupta, Siddharth论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAEwen, Joshua B.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Psychol & Brain Sci, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAHong, Manisha论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ Hosp, Invest Drug Serv, Dept Pharm, Baltimore, MD 21287 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAKratz, Lisa论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Pediat, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAKelley, Richard论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Pediat, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USATierney, Elaine论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Psychiat, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAVaurio, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Psychol, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USABibat, Genila论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Neurogenet Dept, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USASanyal, Abanti论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Bloomberg Sch Publ Hlth, Johns Hopkins Biostat Ctr, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAYenokyan, Gayane论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Bloomberg Sch Publ Hlth, Johns Hopkins Biostat Ctr, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USABrereton, Nga论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Kennedy Krieger Inst, Baltimore, MD USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USAJohnston, Michael V.论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USANaidu, Sakkubai论文数: 0 引用数: 0 h-index: 0机构: Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA
- [9] Trofinetide treatment for Rett syndrome: Lessons to learnMED, 2024, 5 (10): : 1194 - 1196Tropea, Daniela论文数: 0 引用数: 0 h-index: 0机构: Trinity Coll Dublin, St James Hosp, Trinity Translat Med Inst, Trinity Ctr Hlth Sci,Sch Med,Dept Psychiat, Dublin D08W9RT, Ireland Trinity Coll Dublin, Trinity Coll Inst Neurosci, Lloyd Bldg, Dublin, Ireland FutureNeuro, SFI Res Ctr Chron & Rare Neurol Dis, Dublin, Ireland Trinity Coll Dublin, St James Hosp, Trinity Translat Med Inst, Trinity Ctr Hlth Sci,Sch Med,Dept Psychiat, Dublin D08W9RT, Ireland
- [10] Trofinetide for the treatment of Rett syndrome: a randomized phase 3 studyNature Medicine, 2023, 29 : 1468 - 1475Jeffrey L. Neul论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterAlan K. Percy论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterTimothy A. Benke论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterElizabeth M. Berry-Kravis论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterDaniel G. Glaze论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterEric D. Marsh论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterTim Lin论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterSerge Stankovic论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterKathie M. Bishop论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy CenterJames M. Youakim论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt University Medical Center,Vanderbilt Kennedy Center