共 39 条
[1]
Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study
[J].
Acsadi, Gyula
;
Crawford, Thomas O.
;
Mueller-Felber, Wolfgang
;
Shieh, Perry B.
;
Richardson, Randal
;
Natarajan, Niranjana
;
Castro, Diana
;
Ramirez-Schrempp, Daniela
;
Gambino, Giulia
;
Sun, Peng
;
Farwell, Wildon
.
MUSCLE & NERVE,
2021, 63 (05)
:668-677

Acsadi, Gyula
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT 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 Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Mueller-Felber, Wolfgang
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Munich, Dept Neuropediat Dev Neurol & Social Pediat, LMU Campus Innenstadt, Munich, Germany Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Shieh, Perry B.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Calif Los Angeles, Dept Neurol, Clin & Translat Res Ctr, Los Angeles, CA 90024 USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Richardson, Randal
论文数: 0 引用数: 0
h-index: 0
机构:
Gillette Childrens Specialty Healthcare, Pediat Neurol, St Paul, MN USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Natarajan, Niranjana
论文数: 0 引用数: 0
h-index: 0
机构:
Seattle Childrens Res Inst, Dept Neurol, Seattle, WA USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Castro, Diana
论文数: 0 引用数: 0
h-index: 0
机构:
UT Southwestern Med Ctr, Dept Pediat Neurol & Neurotherapeut, Dallas, TX USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Ramirez-Schrempp, Daniela
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen Inc, 14 Cambridge Ctr, Cambridge, MA 02142 USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Gambino, Giulia
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Maidenhead, Berks, England Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Sun, Peng
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen Inc, 14 Cambridge Ctr, Cambridge, MA 02142 USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA

Farwell, Wildon
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen Inc, 14 Cambridge Ctr, Cambridge, MA 02142 USA Univ Connecticut, Sch Med, Connecticut Childrens Med Ctr, Div Pediat Neurol, Farmington, CT USA
[2]
[Anonymous], 2014, REVMAN VERS 5 3 COMP
[3]
MOTOR MILESTONE ASSESSMENT OF INFANTS WITH SPINAL MUSCULAR ATROPHY USING THE HAMMERSMITH INFANT NEUROLOGICAL EXAM-PART 2: EXPERIENCE FROM A NUSINERSEN CLINICAL STUDY
[J].
Bishop, Kathie M.
;
Montes, Jacqueline
;
Finkel, Richard S.
.
MUSCLE & NERVE,
2018, 57 (01)
:142-146

Bishop, Kathie M.
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut Inc, Carlsbad, CA USA Ionis Pharmaceut Inc, Carlsbad, CA USA

Montes, Jacqueline
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Med Ctr, Dept Rehabil, New York, NY USA
Columbia Univ, Med Ctr, Dept Regenerat Med, New York, NY USA
Columbia Univ, Med Ctr, Dept Neurol, New York, NY USA Ionis Pharmaceut Inc, Carlsbad, CA 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 Ionis Pharmaceut Inc, Carlsbad, CA USA
[4]
Copy Number Variations in the Survival Motor Neuron Genes: Implications for Spinal Muscular Atrophy and Other Neurodegenerative Diseases
[J].
Butchbach, Matthew E. R.
.
FRONTIERS IN MOLECULAR BIOSCIENCES,
2016, 3

Butchbach, Matthew E. R.
论文数: 0 引用数: 0
h-index: 0
机构:
Nemours Alfred I duPont Hosp Children, Ctr Appl Clin Genom, Nemours Biomed Res, Wilmington, DE 19803 USA
Nemours Alfred I duPont Hosp Children, Ctr Pediat Res, Nemours Biomed Res, Wilmington, DE 19803 USA
Univ Delaware, Dept Biol Sci, Newark, DE 19716 USA
Thomas Jefferson Univ, Dept Pediat, Philadelphia, PA 19107 USA Nemours Alfred I duPont Hosp Children, Ctr Appl Clin Genom, Nemours Biomed Res, Wilmington, DE 19803 USA
[5]
Age related treatment effect in type II Spinal Muscular Atrophy pediatric patients treated with nusinersen
[J].
Coratti, Giorgia
;
Pane, Marika
;
Lucibello, Simona
;
Pera, Maria Carmela
;
Pasternak, Amy
;
Montes, Jacqueline
;
Sansone, Valeria A.
;
Duong, Tina
;
Young, Sally Dunaway
;
Messina, Sonia
;
D'Amico, Adele
;
Civitello, Matthew
;
Glanzman, Allan M.
;
Bruno, Claudio
;
Salmin, Francesca
;
Tacchetti, Paola
;
Carnicella, Sara
;
Sframeli, Maria
;
Antonaci, Laura
;
Frongia, Anna Lia
;
De Vivo, Darryl C.
;
Darras, Basil T.
;
Day, John
;
Bertini, Enrico
;
Muntoni, Francesco
;
Finkel, Richard
;
Mercuri, Eugenio
.
NEUROMUSCULAR DISORDERS,
2021, 31 (07)
:596-602

Coratti, Giorgia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Pane, Marika
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Lucibello, Simona
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Pera, Maria Carmela
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Pasternak, Amy
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Med Sch, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Montes, Jacqueline
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol, Irving Med Ctr, New York, NY USA
Columbia Univ, Dept Pediat, Irving Med Ctr, New York, NY 10027 USA
Columbia Univ, Dept Rehabil & Regenerat Med, Irving Med Ctr, New York, NY USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Sansone, Valeria A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Milan, Niguarda Hosp, Neuromuscular Omnictr Clin Ctr, Neurorehabil Unit, Milan, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Duong, Tina
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Neurol, Stanford, CA 94305 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Young, Sally Dunaway
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Neurol, Stanford, CA 94305 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

论文数: 引用数:
h-index:
机构:

D'Amico, Adele
论文数: 0 引用数: 0
h-index: 0
机构:
IRCCS Bambino Gesu Childrens Hosp, Dept Neurosci, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Civitello, Matthew
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cent Florida, Coll Med, Nemours Childrens Hosp, Orlando, FL 32816 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Glanzman, Allan M.
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Philadelphia, Dept Phys Therapy, Philadelphia, PA 19104 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Bruno, Claudio
论文数: 0 引用数: 0
h-index: 0
机构:
IRCCS Ist Giannina Gaslini, Ctr Expt & Translat Myol, Genoa, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Salmin, Francesca
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Milan, Niguarda Hosp, Neuromuscular Omnictr Clin Ctr, Neurorehabil Unit, Milan, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Tacchetti, Paola
论文数: 0 引用数: 0
h-index: 0
机构:
IRCCS Ist Giannina Gaslini, Ctr Expt & Translat Myol, Genoa, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Carnicella, Sara
论文数: 0 引用数: 0
h-index: 0
机构:
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Sframeli, Maria
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Messina, Dept Clin & Expt Med, Messina, Italy
Univ Messina, Centm Clin Nemo Sud, Messina, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Antonaci, Laura
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Frongia, Anna Lia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

De Vivo, Darryl C.
论文数: 0 引用数: 0
h-index: 0
机构:
Columbia Univ, Dept Neurol, Irving Med Ctr, New York, NY USA
Columbia Univ, Dept Pediat, Irving Med Ctr, New York, NY 10027 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Darras, Basil T.
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Med Sch, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Day, John
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Neurol, Stanford, CA 94305 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Bertini, Enrico
论文数: 0 引用数: 0
h-index: 0
机构:
IRCCS Bambino Gesu Childrens Hosp, Dept Neurosci, Unit Neuromuscular & Neurodegenerat Disorders, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Muntoni, Francesco
论文数: 0 引用数: 0
h-index: 0
机构:
NIHR Great Ormond St Hosp, Biomed Res Ctr, London, England
UCL Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
Great Ormond St Hosp Sick Children, London, England Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Finkel, Richard
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cent Florida, Coll Med, Nemours Childrens Hosp, Orlando, FL 32816 USA
St Jude Childrens Res Hosp, 332 N Lauderdale St, Memphis, TN 38105 USA Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy

Mercuri, Eugenio
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
Fdn Policlin Univ Agostino Gemelli IRCCS, Ctr Clin Nemo, Rome, Italy Univ Cattolica Sacro Cuore, Pediat Neurol, Rome, Italy
[6]
Detection of novel mutations in the SMN Tudor domain in type ISMA patients
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Cuscó, I
;
Barceló, MJ
;
del Río, E
;
Baiget, M
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Tizzano, EF
.
NEUROLOGY,
2004, 63 (01)
:146-149

Cuscó, I
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain

Barceló, MJ
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain

del Río, E
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain

Baiget, M
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain

Tizzano, EF
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain Univ Hosp Sant Pau, Dept Genet, Barcelona, Spain
[7]
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
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Swoboda, Kathryn J.
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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
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Bias in meta-analysis detected by a simple, graphical test
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Egger, M
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Egger, M
论文数: 0 引用数: 0
h-index: 0
机构:
UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND

Smith, GD
论文数: 0 引用数: 0
h-index: 0
机构:
UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND

Schneider, M
论文数: 0 引用数: 0
h-index: 0
机构:
UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND

Minder, C
论文数: 0 引用数: 0
h-index: 0
机构:
UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND UNIV BERN, DEPT SOCIAL & PREVENT MED, CH-3012 BERN, SWITZERLAND
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The Genetics of Spinal Muscular Atrophy: Progress and Challenges
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Farrar, Michelle A.
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Kiernan, Matthew C.
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NEUROTHERAPEUTICS,
2015, 12 (02)
:290-302

Farrar, Michelle A.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ New S Wales, UNSW Med, Sch Womens & Childrens Hlth, Discipline Paediat, Sydney, NSW, Australia
Neurosci Res Australia, Randwick, NSW, Australia
Sydney Childrens Hosp, Dept Neurol, Randwick, NSW 2031, Australia Univ New S Wales, UNSW Med, Sch Womens & Childrens Hlth, Discipline Paediat, Sydney, NSW, Australia

Kiernan, Matthew C.
论文数: 0 引用数: 0
h-index: 0
机构:
Neurosci Res Australia, Randwick, NSW, Australia
Univ Sydney, Brain & Mind Res Inst, Sydney, NSW 2006, Australia Univ New S Wales, UNSW Med, Sch Womens & Childrens Hlth, Discipline Paediat, Sydney, NSW, Australia
[10]
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