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- [32] Phase 1/2 Open-label Dose Evaluation Study of AXO-Lenti-PD Gene Therapy for Parkinson's Disease: Efficacy, Safety, and Tolerability Data from the Second Cohort at 6 MonthsMOVEMENT DISORDERS, 2021, 36 : S167 - S167Corcoran, G.论文数: 0 引用数: 0 h-index: 0Karunakara, A.论文数: 0 引用数: 0 h-index: 0Vaughn, B.论文数: 0 引用数: 0 h-index: 0Brand-Schieber, E.论文数: 0 引用数: 0 h-index: 0Foltynie, T.论文数: 0 引用数: 0 h-index: 0Barker, R.论文数: 0 引用数: 0 h-index: 0Palfi, S.论文数: 0 引用数: 0 h-index: 0
- [33] Sustained Efficacy and Safety at Week 52 and Up to Three Years in Adults with Glycogen Storage Disease Type iA (GSDIa): Results from a Phase 1/2 Clinical Trial of DTX401, an AAV8-mediated, Liver-Directed Gene TherapyMOLECULAR THERAPY, 2022, 30 (04) : 564 - 564Riba-Wolman, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Farmington, CT USA Univ Connecticut, Farmington, CT USARodriguez-Buritica, David F.论文数: 0 引用数: 0 h-index: 0机构: Univ Texas McGovern Med Sch, Houston, TX USA Univ Connecticut, Farmington, CT USAAhmad, Ayesha论文数: 0 引用数: 0 h-index: 0机构: Univ Michigan, Ann Arbor, MI 48109 USA Univ Connecticut, Farmington, CT USAPico, Maria-Luz Couce论文数: 0 引用数: 0 h-index: 0机构: Hosp Clinico Univ Santiago de Compostela, Santiago De Compostela, Spain Univ Connecticut, Farmington, CT USADerks, Terry G.论文数: 0 引用数: 0 h-index: 0机构: Univ Groningen, Groningen, Netherlands Univ Connecticut, Farmington, CT USAMitchell, John论文数: 0 引用数: 0 h-index: 0机构: Montreal Childrens Hosp, Montreal, PQ, Canada Univ Connecticut, Farmington, CT USAWeinstein, David A.论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Farmington, CT USA Univ Connecticut, Farmington, CT USAMitragotri, Deepali论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Pharmaceut Inc, Cambridge, MA USA Univ Connecticut, Farmington, CT USAValayannopoulos, Vassili论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Pharmaceut Inc, Cambridge, MA USA Univ Connecticut, Farmington, CT USACrombez, Eric论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Pharmaceut Inc, Cambridge, MA USA Univ Connecticut, Farmington, CT USA