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- [1] ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findingsGENETICS IN MEDICINE, 2022, 24 (03) : S350 - S350Shieh, Perry论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAKuntz, Nancy论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL USA Univ Calif Los Angeles, Los Angeles, CA USADowling, James论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Los Angeles, CA USAMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABonnemann, Carsten论文数: 0 引用数: 0 h-index: 0机构: NINDS, Neuromuscular & Neurogenet Disorders Childhood Se, NIH, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAFoley, Dimah论文数: 0 引用数: 0 h-index: 0机构: NINDS, Neuromuscular & Neurogenet Disorders Childhood Se, NIH, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USASeferian, Andreea论文数: 0 引用数: 0 h-index: 0机构: I Motion, Inst Myol, Paris, France Univ Calif Los Angeles, Los Angeles, CA USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: MDUK Oxford Neuromuscular Ctr, Oxford, England Univ Calif Los Angeles, Los Angeles, CA USALawlor, Michael论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI USA Univ Calif Los Angeles, Los Angeles, CA USANoursalehi, Mojtaba论文数: 0 引用数: 0 h-index: 0机构: Formerly Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Formerly Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USARico, Salvador论文数: 0 引用数: 0 h-index: 0机构: Formerly Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAMiller, Weston论文数: 0 引用数: 0 h-index: 0机构: Formerly Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USA
- [2] ASPIRO gene therapy trial in X-linked myotubular myopathy (XLMTM): update on preliminary efficacy and safety findingsNEUROMUSCULAR DISORDERS, 2021, 31 : S47 - S47Shieh, P.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAKuntz, N.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chica, Chicago, IL USA Univ Calif Los Angeles, Los Angeles, CA USADowling, J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Los Angeles, CA USAMueller-Felber, W.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABlaschek, A.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABoennemann, C.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Child, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAFoley, R.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Child, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USASaade, D.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Child, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USASeferian, A.论文数: 0 引用数: 0 h-index: 0机构: Inst Myol, I Motion, Paris, France Univ Calif Los Angeles, Los Angeles, CA USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: MDUK Oxford Neuromuscular Ctr, Oxford, England Univ Calif Los Angeles, Los Angeles, CA USALawlor, M.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Univ Calif Los Angeles, Los Angeles, CA USANoursalehi, M.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAPrasad, S.论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USARico, S.论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAMiller, W.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USA
- [3] Aspiro Phase 1/2 Gene Therapy Trial in X-Linked Myotubular Myopathy (Xlmtm): Preliminary Safety and Efficacy FindingsMOLECULAR THERAPY, 2018, 26 (05) : 4 - 4Kuntz, Nancy论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAShieh, Perry B.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USASmith, Barbara论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USABonnemann, Carsten G.论文数: 0 引用数: 0 h-index: 0机构: NIH, Porter Neurosci Res Ctr, Bldg 10, Bethesda, MD 20892 USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USADowling, James J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USALawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USANoursalehi, Mo论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USARico, Sal论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, Paris, France Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USAPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA
- [4] ASPIRO Phase 1/2 Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety and Efficacy FindingsMOLECULAR THERAPY, 2019, 27 (04) : 306 - 306Shieh, Perry B.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USAKuntz, Nancy论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USASmith, Barbara论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USABonnemann, Carsten G.论文数: 0 引用数: 0 h-index: 0机构: NIH, Porter Neurosci Res Ctr, Bldg 10, Bethesda, MD 20892 USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USADowling, James J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USALawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USAMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USANoursalehi, Mo论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USARico, Salvador论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, Paris, France Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USAPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Med Ctr, Neurol, Los Angeles, CA 90024 USA
- [5] Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: Preliminary results from ASPIRONEUROMUSCULAR DISORDERS, 2022, 32 : S44 - S44Shieh, P.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAKuntz, N.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chica, Chicago, IL USA Univ Calif Los Angeles, Los Angeles, CA USADowling, J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Los Angeles, CA USAMueller-Felber, W.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABlaschek, A.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABonnemann, C.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Child, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAFoley, R.论文数: 0 引用数: 0 h-index: 0机构: Neuromuscular & Neurogenet Disorders Child, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USA论文数: 引用数: h-index:机构:Seferian, A.论文数: 0 引用数: 0 h-index: 0机构: Inst Myol, I Mot, Paris, France Univ Calif Los Angeles, Los Angeles, CA USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: MDUK Oxford Neuromuscular Ctr, Oxford, England Univ Calif Los Angeles, Los Angeles, CA USABowden, A.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USASarazen, M.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USACoats, J.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USALakshman, N.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAHan, C.论文数: 0 引用数: 0 h-index: 0机构: Astellas Pharma Global Dev, Northbrook, IL USA Univ Calif Los Angeles, Los Angeles, CA USAPrasad, S.论文数: 0 引用数: 0 h-index: 0机构: Formerly Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USARico, S.论文数: 0 引用数: 0 h-index: 0机构: Formerly Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAMiller, W.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USA
- [6] X-Linked myotubular myopathy (XLMTM): phenotypic variabilityNEUROMUSCULAR DISORDERS, 2017, 27 : S170 - S170Ortez, C.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainNatera, D.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainColomer, J.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainItzep, D.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainAlarcon, M.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainFrongia, A.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainJou, C.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainCodina, A.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainJimenez-Mallebrera, C.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainMartorell, L.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainBiancalana, V.论文数: 0 引用数: 0 h-index: 0机构: Fac Med C HRU, Strasbourg, France Hosp St Joan Deu UB, Barcelona, SpainGonzalez, L.论文数: 0 引用数: 0 h-index: 0机构: Hosp Santa Creu & Sant Pau, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainGallano, P.论文数: 0 引用数: 0 h-index: 0机构: Hosp Santa Creu & Sant Pau, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, SpainNascimento, A.论文数: 0 引用数: 0 h-index: 0机构: Hosp St Joan Deu UB, Barcelona, Spain Hosp St Joan Deu UB, Barcelona, Spain
- [7] ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-TreatmentNEUROLOGY, 2020, 94 (15)Shieh, Perry B.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Univ Calif Los Angeles, Los Angeles, CA USAKuntz, Nancy论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Univ Calif Los Angeles, Los Angeles, CA USASmith, Barbara论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL 32611 USA Univ Calif Los Angeles, Los Angeles, CA USADowling, James论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Los Angeles, CA USAMueller-Felber, Wolfgang论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USABoennemann, Carsten G.论文数: 0 引用数: 0 h-index: 0机构: NIH Porter Neuroscience Res Ctr, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAServais, Laurent论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, Paris, France Univ Calif Los Angeles, Los Angeles, CA USAMuntoni, Francesco论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Hosp Sick Children, London, England Univ Calif Los Angeles, Los Angeles, CA USABlaschek, Astrid论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA USANeuhaus, Sarah论文数: 0 引用数: 0 h-index: 0机构: NIH Porter Neuroscience Res Ctr, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAAlfano, Lindsay N.论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Univ Calif Los Angeles, Los Angeles, CA USABeggs, Alan H.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Boston, Boston, MA USA Univ Calif Los Angeles, Los Angeles, CA USABuj-Bello, Ana论文数: 0 引用数: 0 h-index: 0机构: Genethon, Evry, France Univ Calif Los Angeles, Los Angeles, CA USAChilders, Martin论文数: 0 引用数: 0 h-index: 0机构: AskBio, New York, NY USA Univ Calif Los Angeles, Los Angeles, CA USA论文数: 引用数: h-index:机构:Graham, Robert J.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Boston, Boston, MA USA Univ Calif Los Angeles, Los Angeles, CA USAJain, Minal论文数: 0 引用数: 0 h-index: 0机构: NIH Hatfield Clin Res Ctr, Bethesda, MD USA Univ Calif Los Angeles, Los Angeles, CA USAJames, Emma S.论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USALawlor, Michael W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI USA Univ Calif Los Angeles, Los Angeles, CA USALee, Jun论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAMacBean, Victoria论文数: 0 引用数: 0 h-index: 0机构: Brunel Univ London, Uxbridge, Middx, England Univ Calif Los Angeles, Los Angeles, CA USAMavilio, Fulvio论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAMurtagh, Michael论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USANoursalehi, Mojtaba论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USAPrasad, Suyash论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USARico, Salvador论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA USA
- [8] ASPIRO Gene therapy trial in X-linked myotubular myopathy (XLMTM): Update on preliminary safety and efficacy findings up to 72 weeks post-treatmentNEUROMUSCULAR DISORDERS, 2020, 30 : S65 - S66Shieh, P.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA 90024 USA Univ Calif Los Angeles, Los Angeles, CA 90024 USAKuntz, N.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp, Chicago, IL USA Univ Calif Los Angeles, Los Angeles, CA 90024 USABonnemann, C.论文数: 0 引用数: 0 h-index: 0机构: NIH, Bldg 10, Bethesda, MD 20892 USA Univ Calif Los Angeles, Los Angeles, CA 90024 USAMueller-Felber, W.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ, Munich, Germany Univ Calif Los Angeles, Los Angeles, CA 90024 USALawlor, M. W.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Univ Calif Los Angeles, Los Angeles, CA 90024 USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: Hop Armand Trousseau, Paris, France Univ Calif Los Angeles, Los Angeles, CA 90024 USASmith, B.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL USA Univ Calif Los Angeles, Los Angeles, CA 90024 USANoursalehi, M.论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA 90024 USAPrasad, S.论文数: 0 引用数: 0 h-index: 0机构: Audentes Therapeut, San Francisco, CA USA Univ Calif Los Angeles, Los Angeles, CA 90024 USADowling, J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Univ Calif Los Angeles, Los Angeles, CA 90024 USA
- [9] Improved Respiratory Outcomes for X-Linked Myotubular Myopathy (XLMTM) with Gene Replacement Therapy, Resamirigene Bilparvovec (ASPIRO): Preliminary Results from ASPIRO, a Phase 1/2/3 StudyAMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE, 2022, 205Perez, G. F.论文数: 0 引用数: 0 h-index: 0机构: Oishei Childrens Hosp, Pulm, Buffalo, NY USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAShieh, P.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif Los Angeles, Los Angeles, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAKuntz, N.论文数: 0 引用数: 0 h-index: 0机构: Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USADowling, J.论文数: 0 引用数: 0 h-index: 0机构: Hosp Sick Children, Toronto, ON, Canada Oishei Childrens Hosp, Pulm, Buffalo, NY USAMueller-Felber, W.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Oishei Childrens Hosp, Pulm, Buffalo, NY USABlaschek, A.论文数: 0 引用数: 0 h-index: 0机构: Klinikum Univ Munchen, Munich, Germany Oishei Childrens Hosp, Pulm, Buffalo, NY USABonnemann, C.论文数: 0 引用数: 0 h-index: 0机构: NINDS, Neuromuscular & Neurogenet Disorders Childhood Se, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAFoley, A.论文数: 0 引用数: 0 h-index: 0机构: NINDS, Neuromuscular & Neurogenet Disorders Childhood Se, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USASaade, D.论文数: 0 引用数: 0 h-index: 0机构: NINDS, Neuromuscular & Neurogenet Disorders Childhood Se, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USASeferian, A.论文数: 0 引用数: 0 h-index: 0机构: Inst Myol, I Mot, Paris, France Oishei Childrens Hosp, Pulm, Buffalo, NY USAServais, L.论文数: 0 引用数: 0 h-index: 0机构: MDUK Oxford Neuromuscular Ctr, Oxford, England Oishei Childrens Hosp, Pulm, Buffalo, NY USALawlor, M.论文数: 0 引用数: 0 h-index: 0机构: Med Coll Wisconsin, Milwaukee, WI 53226 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAMacBean, V.论文数: 0 引用数: 0 h-index: 0机构: Brunel Univ London, Dept Hlth Sci, London, England Oishei Childrens Hosp, Pulm, Buffalo, NY USACoats, J.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAHan, C.论文数: 0 引用数: 0 h-index: 0机构: Astellas Pharma Global Dev, Northbrook, IL USA Oishei Childrens Hosp, Pulm, Buffalo, NY USALee, J.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USANoursalehi, M.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAPrasad, S.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USARico, S.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAMiller, W.论文数: 0 引用数: 0 h-index: 0机构: Astellas Gene Therapies, San Francisco, CA USA Oishei Childrens Hosp, Pulm, Buffalo, NY USAGraham, R.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA Oishei Childrens Hosp, Pulm, Buffalo, NY USA
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