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[1]
[Anonymous], 2018, INF POS TER NUS SPIN
[2]
Castro D., 2020, Neurology, V94
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New and Developing Therapies in Spinal Muscular Atrophy: From Genotype to Phenotype to Treatment and Where Do We Stand?
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Chen, Tai-Heng
.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES,
2020, 21 (09)

Chen, Tai-Heng
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Southern Calif, Dept Biol Sci, Sect Neurobiol, Los Angeles, CA 90089 USA
Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Dept Pediat, Div Pediat Emergency, Kaohsiung 80708, Taiwan
Kaohsiung Med Univ, Grad Inst Clin Med, PhD Program Translat Med, Taipei 11529, Taiwan
Acad Sinica, Taipei 11529, Taiwan Univ Southern Calif, Dept Biol Sci, Sect Neurobiol, Los Angeles, CA 90089 USA
[4]
Chiriboga CA, 2020, NEUROLOGY, V94
[5]
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
[J].
De Vivo, Darryl C.
;
Bertini, Enrico
;
Swoboda, Kathryn J.
;
Hwu, Wuh-Liang
;
Crawford, Thomas O.
;
Finkel, Richard S.
;
Kirschner, Janbernd
;
Kuntz, Nancy L.
;
Parsons, Julie A.
;
Ryan, Monique M.
;
Butterfield, Russell J.
;
Topaloglu, Haluk
;
Ben-Omran, Tawfeg
;
Sansone, Valeria A.
;
Jong, Yuh-Jyh
;
Shu, Francy
;
Staropoli, John F.
;
Kerr, Douglas
;
Sandrock, Alfred W.
;
Stebbins, Christopher
;
Petrillo, Marco
;
Braley, Gabriel
;
Johnson, Kristina
;
Foster, Richard
;
Gheuens, Sarah
;
Bhan, Ishir
;
Reyna, Sandra P.
;
Fradette, Stephanie
;
Farwell, Wildon
.
NEUROMUSCULAR DISORDERS,
2019, 29 (11)
:842-856

De Vivo, Darryl C.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Bertini, Enrico
论文数: 0 引用数: 0
h-index: 0
机构:
IRCCS, Unit Neuromuscular & Neurodegenerat Disorders, Postgrad Bambino Gesu Childrens Res Hosp, Rome, Italy Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Swoboda, Kathryn J.
论文数: 0 引用数: 0
h-index: 0
机构:
Massachusetts Gen Hosp, Dept Neurol, Ctr Genom Med, Boston, MA 02114 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Hwu, Wuh-Liang
论文数: 0 引用数: 0
h-index: 0
机构:
Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, Taipei, Taiwan Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Crawford, Thomas O.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21205 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Finkel, Richard S.
论文数: 0 引用数: 0
h-index: 0
机构:
Nemours Childrens Hosp, Dept Pediat, Div Neurol, Orlando, FL USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Kirschner, Janbernd
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany
Univ Med Hosp, Dept Neuropediat, Bonn, Germany Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Kuntz, Nancy L.
论文数: 0 引用数: 0
h-index: 0
机构:
Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Parsons, Julie A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Colorado, Childrens Hosp Colorado, Sch Med, Aurora, CO USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Ryan, Monique M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Melbourne, Royal Childrens Hosp, Murdoch Childrens Res Inst, Melbourne, Vic, Australia Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Butterfield, Russell J.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Utah, Dept Pediat & Neurol, Salt Lake City, UT USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Topaloglu, Haluk
论文数: 0 引用数: 0
h-index: 0
机构:
Hacettepe Univ, Dept Pediat Neurol, Ankara, Turkey Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Ben-Omran, Tawfeg
论文数: 0 引用数: 0
h-index: 0
机构:
Qatar Fdn, Dept Pediat, Sidra Med, Doha, Qatar
Hamad Med Corp, Div Clin & Metab Genet, Dept Pediat, Doha, Qatar Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Sansone, Valeria A.
论文数: 0 引用数: 0
h-index: 0
机构:
NEMO Clin Ctr NEuroMuscular Omniserv, Milan, Italy
Univ Milan, Dept Biomed Sci Hlth, Milan, Italy Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Jong, Yuh-Jyh
论文数: 0 引用数: 0
h-index: 0
机构:
Kaohsiung Med Univ, Kaohsiung Med Univ Hosp, Dept Pediat & Lab Med, Coll Med, Kaohsiung, Taiwan Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Shu, Francy
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90095 USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Staropoli, John F.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Kerr, Douglas
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Sandrock, Alfred W.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Stebbins, Christopher
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Petrillo, Marco
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Braley, Gabriel
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Johnson, Kristina
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Foster, Richard
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Maidenhead, Berks, England Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Gheuens, Sarah
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Bhan, Ishir
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Reyna, Sandra P.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Fradette, Stephanie
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA

Farwell, Wildon
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Columbia Univ, Dept Neurol & Pediat, Irving Med Ctr, New York, NY 10032 USA
[6]
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
[J].
Finkel, R. S.
;
Mercuri, E.
;
Darras, B. T.
;
Connolly, A. M.
;
Kuntz, N. L.
;
Kirschner, J.
;
Chiriboga, C. A.
;
Saito, K.
;
Servais, L.
;
Tizzano, E.
;
Topaloglu, H.
;
Tulinius, M.
;
Montes, J.
;
Glanzman, A. M.
;
Bishop, K.
;
Zhong, Z. J.
;
Gheuens, S.
;
Bennett, C. F.
;
Schneider, E.
;
Farwell, W.
;
De Vivo, D. C.
.
NEW ENGLAND JOURNAL OF MEDICINE,
2017, 377 (18)
:1723-1732

Finkel, R. S.
论文数: 0 引用数: 0
h-index: 0
机构:
Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Mercuri, E.
论文数: 0 引用数: 0
h-index: 0
机构:
Catholic Univ, Dept Pediat Neurol, Rome, Italy Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Darras, B. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Boston Childrens Hosp, Dept Neurol, Boston, MA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Connolly, A. M.
论文数: 0 引用数: 0
h-index: 0
机构:
St Louis Childrens Hosp, Dept Neurol, St Louis, MO 63178 USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Kuntz, N. L.
论文数: 0 引用数: 0
h-index: 0
机构:
Ann & Robert H Lurie Childrens Hosp Chicago, Dept Pediat, Chicago, IL 60611 USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Kirschner, J.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Freiburg, Fac Med, Med Ctr, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Chiriboga, C. A.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol, New York, NY USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Saito, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Tokyo Womens Med Coll, Inst Med Genet, Tokyo, Japan
Tokyo Womens Med Coll, Dept Pediat, Tokyo, Japan Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Servais, L.
论文数: 0 引用数: 0
h-index: 0
机构:
Inst Mot, Paris, France Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Tizzano, E.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Vall dHebron, Dept Clin & Mol Genet, Barcelona, Spain
Univ Hosp Vall dHebron, Rare Dis Unit, Barcelona, Spain
Ctr Invest Biomed Enfermedades Raras CIBERER, Barcelona, Spain Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Topaloglu, H.
论文数: 0 引用数: 0
h-index: 0
机构:
Hacettepe Univ, Sch Med, Dept Pediat, Ankara, Turkey Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Tulinius, M.
论文数: 0 引用数: 0
h-index: 0
机构:
Gothenburg Univ, Queen Silvia Childrens Hosp, Dept Pediat, Gothenburg, Sweden Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Montes, J.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol, New York, NY USA
Columbia Univ, Dept Rehabil & Regenerat Med, New York, NY USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Glanzman, A. M.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Dept Phys Therapy, Philadelphia, PA 19104 USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Bishop, K.
论文数: 0 引用数: 0
h-index: 0
机构:
lonis Pharmaceut, Carlsbad, CA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Zhong, Z. J.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Gheuens, S.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Bennett, C. F.
论文数: 0 引用数: 0
h-index: 0
机构:
lonis Pharmaceut, Carlsbad, CA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Schneider, E.
论文数: 0 引用数: 0
h-index: 0
机构:
lonis Pharmaceut, Carlsbad, CA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

Farwell, W.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA

De Vivo, D. C.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol, Med Ctr, New York, NY USA
Columbia Univ, Dept Pediat, Med Ctr, New York, NY 10027 USA Nemours Childrens Hosp, Dept Pediat, Div Neurol, 13535 Nemours Pkwy,5th Fl, Orlando, FL 32827 USA
[7]
Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study
[J].
Hagenacker, Tim
;
Wurster, Claudia D.
;
Guenther, Rene
;
Schreiber-Katz, Olivia
;
Osmanovic, Alma
;
Petri, Susanne
;
Weiler, Markus
;
Ziegler, Andreas
;
Kuttler, Josua
;
Koch, Jan C.
;
Schneider, Ilka
;
Wunderlich, Gilbert
;
Schloss, Natalie
;
Lehmann, Helmar C.
;
Cordts, Isabell
;
Deschauer, Marcus
;
Lingor, Paul
;
Kamm, Christoph
;
Stolte, Benjamin
;
Pietruck, Lena
;
Totzeck, Andreas
;
Kizina, Kathrin
;
Monninghoff, Christoph
;
von Velsen, Otgonzul
;
Ose, Claudia
;
Reichmann, Heinz
;
Forsting, Michael
;
Pechmann, Astrid
;
Kirschner, Janbernd
;
Ludolph, Albert C.
;
Hermann, Andreas
;
Kleinschnitz, Christoph
.
LANCET NEUROLOGY,
2020, 19 (04)
:317-325

Hagenacker, Tim
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Wurster, Claudia D.
论文数: 0 引用数: 0
h-index: 0
机构:
Ulm Univ, Dept Neurol, Ulm, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Guenther, Rene
论文数: 0 引用数: 0
h-index: 0
机构:
Tech Univ Dresden, Univ Klinikum Carl Gustav Carus, Dept Neurol, Dresden, Germany
German Ctr Neurodegenerat Dis Dresden, Dresden, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Schreiber-Katz, Olivia
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, Dept Neurol, Hannover, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Osmanovic, Alma
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, Dept Neurol, Hannover, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Petri, Susanne
论文数: 0 引用数: 0
h-index: 0
机构:
Hannover Med Sch, Dept Neurol, Hannover, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Weiler, Markus
论文数: 0 引用数: 0
h-index: 0
机构:
Heidelberg Univ Hosp, Dept Neurol, Heidelberg, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Ziegler, Andreas
论文数: 0 引用数: 0
h-index: 0
机构:
Heidelberg Univ Hosp, Ctr Child & Adolescent Med, Div Child Neurol & Metab Med, Heidelberg, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Kuttler, Josua
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Gottingen, Dept Neurol, Gottingen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Koch, Jan C.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Med Gottingen, Dept Neurol, Gottingen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Schneider, Ilka
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Halle Saale, Dept Neurol, Halle, Saale, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Wunderlich, Gilbert
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cologne, Fac Med, Dept Neurol, Cologne, Germany
Univ Cologne, Fac Med, Ctr Rare Dis, Cologne, Germany
Univ Cologne, Univ Hosp Cologne, Cologne, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Schloss, Natalie
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cologne, Fac Med, Dept Neurol, Cologne, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Lehmann, Helmar C.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cologne, Fac Med, Dept Neurol, Cologne, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Cordts, Isabell
论文数: 0 引用数: 0
h-index: 0
机构:
Tech Univ Munich, Dept Neurol, Klinikum Rechts Isar, Munich, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Deschauer, Marcus
论文数: 0 引用数: 0
h-index: 0
机构:
Tech Univ Munich, Dept Neurol, Klinikum Rechts Isar, Munich, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Lingor, Paul
论文数: 0 引用数: 0
h-index: 0
机构:
Tech Univ Munich, Dept Neurol, Klinikum Rechts Isar, Munich, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

论文数: 引用数:
h-index:
机构:

Stolte, Benjamin
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Pietruck, Lena
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Totzeck, Andreas
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Kizina, Kathrin
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Monninghoff, Christoph
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Diagnost & Intervent Radiol & Neuroradiol, Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

von Velsen, Otgonzul
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Inst Med Informat Biometr & Epidemiol, Essen, Germany
Univ Hosp Essen, Ctr Clin Trials, Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Ose, Claudia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Inst Med Informat Biometr & Epidemiol, Essen, Germany
Univ Hosp Essen, Ctr Clin Trials, Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Reichmann, Heinz
论文数: 0 引用数: 0
h-index: 0
机构:
Tech Univ Dresden, Univ Klinikum Carl Gustav Carus, Dept Neurol, Dresden, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Forsting, Michael
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Diagnost & Intervent Radiol & Neuroradiol, Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Pechmann, Astrid
论文数: 0 引用数: 0
h-index: 0
机构:
Med Ctr Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Kirschner, Janbernd
论文数: 0 引用数: 0
h-index: 0
机构:
Med Ctr Univ Freiburg, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany
Univ Med Ctr Bonn, Dept Neuropediat, Bonn, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Ludolph, Albert C.
论文数: 0 引用数: 0
h-index: 0
机构:
Ulm Univ, Dept Neurol, Ulm, Germany
German Ctr Neurodegenerat Dis Ulm, Ulm, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Hermann, Andreas
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Rostock, Dept Neurol, Rostock, Germany
Univ Rostock, Univ Med Ctr Rostock, Dept Neurol, Translat Neurodegenerat Sect Albrecht Kosser, Rostock, Germany
German Ctr Neurodegenerat Dis Rostock, Rostock, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany

Kleinschnitz, Christoph
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany Univ Hosp Essen, Dept Neurol, D-45127 Essen, Germany
[8]
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
[J].
Mendell, J. R.
;
Al-Zaidy, S.
;
Shell, R.
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Rodino-Klapac, L. R.
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Prior, T. W.
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Lowes, L.
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Alfano, L.
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Berry, K.
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Church, K.
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Kissel, J. T.
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Nagendran, S.
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L'Italien, J.
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Sproule, D. M.
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Wells, C.
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Cardenas, J. A.
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Heitzer, M. D.
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Kaspar, A.
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Corcoran, S.
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Braun, L.
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Likhite, S.
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Miranda, C.
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2017, 377 (18)
:1713-1722

Mendell, J. R.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Al-Zaidy, S.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

论文数: 引用数:
h-index:
机构:

Arnold, W. D.
论文数: 0 引用数: 0
h-index: 0
机构:
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Rodino-Klapac, L. R.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Prior, T. W.
论文数: 0 引用数: 0
h-index: 0
机构:
Ohio State Univ, Dept Pathol, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Lowes, L.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

论文数: 引用数:
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Berry, K.
论文数: 0 引用数: 0
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机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Church, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Kissel, J. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Nagendran, S.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

L'Italien, J.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Sproule, D. M.
论文数: 0 引用数: 0
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AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Wells, C.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Cardenas, J. A.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Heitzer, M. D.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Kaspar, A.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Corcoran, S.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Braun, L.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Likhite, S.
论文数: 0 引用数: 0
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Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Miranda, C.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Meyer, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Foust, K. D.
论文数: 0 引用数: 0
h-index: 0
机构:
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Burghes, A. H. M.
论文数: 0 引用数: 0
h-index: 0
机构:
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA
Ohio State Univ, Dept Mol & Cellular Biochem, Columbus, OH 43210 USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA

Kaspar, B. K.
论文数: 0 引用数: 0
h-index: 0
机构:
Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA
Ohio State Univ, Dept Neurol, Columbus, OH 43210 USA
AveXis, Bannockburn, IL USA Nationwide Childrens Hosp, Res Inst, Ctr Gene Therap, Columbus, OH 43205 USA
[9]
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
[J].
Mercuri, E.
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Iannaccone, S. T.
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Kirschner, J.
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Kuntz, N. L.
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Saito, K.
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Shieh, P. B.
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Tulinius, M.
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Mazzone, E. S.
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Montes, J.
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Bishop, K. M.
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Yang, Q.
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Foster, R.
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Gheuens, S.
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Bennett, C. F.
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Farwell, W.
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Schneider, E.
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De Vivo, D. C.
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Finkel, R. S.
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Bradley, Walter G.
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Kaufmann, Petra
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Dickson, Patricia I.
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Reingold, Stephen C.
;
Davis, Charles S.
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Arredondo, Kristen
;
Castro, Diana
;
Cowie, Margaret
;
Farrow-Gillespie, Alan
;
Hebert, Andrew
;
Kauk, Melissa
;
Miller, Nancy
;
Nelson, Leslie
;
Spain, Thomas, Jr.
;
Cappell, Joshua
;
Constantinescu, Andrei
;
Cruz, Rosangel
;
Dastgir, Jahannaz
;
De Vivo, Darryl
;
Dunaway, Sally
;
Engelstad, Kristin
;
Khandji, Alexander G.
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Kramer, Samantha
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Marra, Jonathan
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Popolizio, Molly
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NEW ENGLAND JOURNAL OF MEDICINE,
2018, 378 (07)
:625-635

Mercuri, E.
论文数: 0 引用数: 0
h-index: 0
机构:
Catholic Univ, Dept Pediat Neurol, Rome, Italy Catholic Univ, Dept Pediat Neurol, Rome, Italy

Darras, B. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Boston Childrens Hosp, Dept Neurol, Boston, MA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Chiriboga, C. A.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ Med Ctr, Dept Neurol, New York, NY USA
Columbia Univ Med Ctr, Dept Pediat, New York, NY USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Day, J. W.
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Sch Med, Dept Neurol, Stanford, CA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Campbell, C.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp, London Hlth Sci Ctr, London, ON, Canada Catholic Univ, Dept Pediat Neurol, Rome, Italy

Connolly, A. M.
论文数: 0 引用数: 0
h-index: 0
机构:
Washington Univ, Sch Med, Dept Neurol, St Louis, MO 63110 USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Iannaccone, S. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, Dallas, TX USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Kirschner, J.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Freiburg, Med Ctr, Fac Med, Dept Neuropediat & Muscle Disorders, Freiburg, Germany Catholic Univ, Dept Pediat Neurol, Rome, Italy

Kuntz, N. L.
论文数: 0 引用数: 0
h-index: 0
机构:
Ann & Robert H Lurie Childrens Hosp Chicago, Dept Pediat, Chicago, IL 60611 USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Saito, K.
论文数: 0 引用数: 0
h-index: 0
机构:
Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan
Tokyo Womens Med Univ, Dept Pediat, Tokyo, Japan Catholic Univ, Dept Pediat Neurol, Rome, Italy

Shieh, P. B.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Los Angeles, David Geffen Sch Med, Los Angeles, CA 90095 USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Tulinius, M.
论文数: 0 引用数: 0
h-index: 0
机构:
Gothenburg Univ, Queen Silvia Childrens Hosp, Dept Pediat, Gothenburg, Sweden Catholic Univ, Dept Pediat Neurol, Rome, Italy

Mazzone, E. S.
论文数: 0 引用数: 0
h-index: 0
机构:
Catholic Univ, Dept Pediat Neurol, Rome, Italy Catholic Univ, Dept Pediat Neurol, Rome, Italy

Montes, J.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ Med Ctr, Dept Neurol, New York, NY USA
Columbia Univ Med Ctr, Dept Rehabil & Regenerat Med, New York, NY USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Bishop, K. M.
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, Carlsbad, CA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Yang, Q.
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, Carlsbad, CA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Foster, R.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Gheuens, S.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Bennett, C. F.
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, Carlsbad, CA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Farwell, W.
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Schneider, E.
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, Carlsbad, CA USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

De Vivo, D. C.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ Med Ctr, Dept Neurol, New York, NY USA
Columbia Univ Med Ctr, Dept Pediat, New York, NY USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Finkel, R. S.
论文数: 0 引用数: 0
h-index: 0
机构:
Nemours Childrens Hosp, Dept Pediat, Div Neurol, Orlando, FL USA Catholic Univ, Dept Pediat Neurol, Rome, Italy

Bradley, Walter G.
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Kaufmann, Petra
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h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Dickson, Patricia I.
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Reingold, Stephen C.
论文数: 0 引用数: 0
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Davis, Charles S.
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Arredondo, Kristen
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Castro, Diana
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Cowie, Margaret
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Farrow-Gillespie, Alan
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Hebert, Andrew
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Kauk, Melissa
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Miller, Nancy
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Nelson, Leslie
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Spain, Thomas, Jr.
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h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Cappell, Joshua
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Constantinescu, Andrei
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Cruz, Rosangel
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Dastgir, Jahannaz
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

De Vivo, Darryl
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Dunaway, Sally
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Engelstad, Kristin
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Khandji, Alexander G.
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Kramer, Samantha
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Marra, Jonathan
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Popolizio, Molly
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Salazar, Rachel
论文数: 0 引用数: 0
h-index: 0
机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy

Weimer, Louis H.
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机构: Catholic Univ, Dept Pediat Neurol, Rome, Italy
[10]
Mercuri E, 2020, SUNFISH 2