共 54 条
[1]
Treatment of spinal muscular atrophy cells with drugs that upregulate SMN expression reveals inter- and intra-patient variability
[J].
Also-Rallo, Eva
;
Alias, Laura
;
Martinez-Hernandez, Rebeca
;
Caselles, Lidia
;
Barcelo, Maria J.
;
Baiget, Montserrat
;
Bernal, Sara
;
Tizzano, Eduardo F.
.
EUROPEAN JOURNAL OF HUMAN GENETICS,
2011, 19 (10)
:1059-1065

Also-Rallo, Eva
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Alias, Laura
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Martinez-Hernandez, Rebeca
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Caselles, Lidia
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Barcelo, Maria J.
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Baiget, Montserrat
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Bernal, Sara
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain

Tizzano, Eduardo F.
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, ISCIII, Barcelona 08041, Spain Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
[2]
Prognostic Factors and Treatment-Effect Modifiers in Spinal Muscular Atrophy
[J].
Baranello, Giovanni
;
Gorni, Ksenija
;
Daigl, Monica
;
Kotzeva, Anna
;
Evans, Rachel
;
Hawkins, Neil
;
Scott, David A.
;
Mahajan, Anadi
;
Muntoni, Francesco
;
Servais, Laurent
.
CLINICAL PHARMACOLOGY & THERAPEUTICS,
2021, 110 (06)
:1435-1454

Baranello, Giovanni
论文数: 0 引用数: 0
h-index: 0
机构:
UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
Ist Neurol Carlo Besta, Dev Neurol Unit, Fdn Ist Ricovero & Cura, Carattere Sci, Milan, Italy UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Gorni, Ksenija
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann Roche Ltd, Basel, Switzerland UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Daigl, Monica
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann Roche Ltd, Basel, Switzerland UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Kotzeva, Anna
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann Roche Ltd, Basel, Switzerland UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Evans, Rachel
论文数: 0 引用数: 0
h-index: 0
机构:
Visible Analyt, Oxford, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Hawkins, Neil
论文数: 0 引用数: 0
h-index: 0
机构:
Bridge Med Consulting Ltd, London, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Scott, David A.
论文数: 0 引用数: 0
h-index: 0
机构:
Visible Analyt, Oxford, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Mahajan, Anadi
论文数: 0 引用数: 0
h-index: 0
机构:
Bridge Med Consulting Ltd, London, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Muntoni, Francesco
论文数: 0 引用数: 0
h-index: 0
机构:
UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
UCL, Natl Inst Hlth Res, Great Ormond St Hosp Natl Hlth Serv Trust, Biomed Res Ctr,Great Ormond St Inst Child Hlth, London, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England

Servais, Laurent
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Liege, Div Child Neurol, Ctr Hosp Reg References Malad Neuromusculaires, Reference Ctr Neuromuscular Dis,Dept Pediat, Liege, Belgium
Univ La Citadelle, Liege, Belgium
Univ Oxford, Dept Paediat, Muscular Dystrophy UK Oxford Neuromuscular Ctr, Oxford, England UCL Great Ormond St Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
[3]
Risdiplam in Type 1 Spinal Muscular Atrophy
[J].
Baranello, Giovanni
;
Darras, Basil T.
;
Day, John W.
;
Deconinck, Nicolas
;
Klein, Andrea
;
Masson, Riccardo
;
Mercuri, Eugenio
;
Rose, Kristy
;
El-Khairi, Muna
;
Gerber, Marianne
;
Gorni, Ksenija
;
Khwaja, Omar
;
Kletzl, Heidemarie
;
Scalco, Renata S.
;
Seabrook, Timothy
;
Fontoura, Paulo
;
Servais, Laurent
.
NEW ENGLAND JOURNAL OF MEDICINE,
2021, 384 (10)
:915-923

Baranello, Giovanni
论文数: 0 引用数: 0
h-index: 0
机构:
UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England
Great Ormond St Hosp Trust, London, England
Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

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 UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Day, John W.
论文数: 0 引用数: 0
h-index: 0
机构:
Stanford Univ, Dept Neurol, Palo Alto, CA 94304 USA UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Deconinck, Nicolas
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Libre Bruxelles, Queen Fabiola Childrens Univ Hosp, Ctr Reference Malad Neuromusculaires, Brussels, Belgium
Univ Ziekenhuis Gent, Neuromuscular Reference Ctr, Ghent, Belgium UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Klein, Andrea
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Childrens Hosp Basel, Div Pediat Neurol, Basel, Switzerland
Univ Bern, Pediat Neurol, Inselspital, Bern, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Masson, Riccardo
论文数: 0 引用数: 0
h-index: 0
机构:
Fdn IRCCS Ist Neurol Carlo Besta, Dev Neurol Unit, Milan, Italy UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Mercuri, Eugenio
论文数: 0 引用数: 0
h-index: 0
机构:
Catholic Univ, Pediat Neurol Inst, Rome, Italy
Fdn Policlin Gemelli IRCCS, Nemo Pediat, Rome, Italy UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Rose, Kristy
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Sydney, Fac Med & Hlth, Discipline Physiotherapy, Sydney, NSW, Australia UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

El-Khairi, Muna
论文数: 0 引用数: 0
h-index: 0
机构:
Roche Prod, Welwyn Garden City, Herts, England UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Gerber, Marianne
论文数: 0 引用数: 0
h-index: 0
机构:
Pharma Dev Safety, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Gorni, Ksenija
论文数: 0 引用数: 0
h-index: 0
机构:
Prod Dev Med Affairs Neurosci & Rare Dis, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Khwaja, Omar
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann La Roche, Basel, Switzerland
Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Kletzl, Heidemarie
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann La Roche, Basel, Switzerland
Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Scalco, Renata S.
论文数: 0 引用数: 0
h-index: 0
机构:
Pharma Dev Neurol, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Seabrook, Timothy
论文数: 0 引用数: 0
h-index: 0
机构:
F Hoffmann La Roche, Basel, Switzerland
Roche Innovat Ctr Basel, Roche Pharmaceut Res & Early Dev, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Fontoura, Paulo
论文数: 0 引用数: 0
h-index: 0
机构:
Prod Dev Med Affairs Neurosci & Rare Dis, Basel, Switzerland UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England

Servais, Laurent
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Oxford, Dept Paediat, Muscular Dystrophy UK Oxford Neuromuscular Ctr, Oxford, England
Univ Hosp Liege, Div Child Neurol, Dept Pediat, Ctr References Malad Neuromusculaires, Liege, Belgium
Univ Liege, Liege, Belgium
Hop Armand Trousseau, Paris, France UCL, Great Ormond St Inst Child Hlth, Great Ormond St Hosp Biomed Res Ctr, Dubowitz Neuromuscular Ctr,Natl Inst Hlth Res, London, England
[4]
Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons
[J].
Boza-Moran, Maria G.
;
Martinez-Hernandez, Rebeca
;
Bernal, Sara
;
Wanisch, Klaus
;
Also-Rallo, Eva
;
Le Heron, Anita
;
Alias, Laura
;
Denis, Cecile
;
Girard, Mathilde
;
Yee, Jiing-Kuan
;
Tizzano, Eduardo F.
;
Yanez-Munoz, Rafael J.
.
SCIENTIFIC REPORTS,
2015, 5

论文数: 引用数:
h-index:
机构:

Martinez-Hernandez, Rebeca
论文数: 0 引用数: 0
h-index: 0
机构:
Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, Barcelona, Spain Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Bernal, Sara
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, Barcelona, Spain Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Wanisch, Klaus
论文数: 0 引用数: 0
h-index: 0
机构:
Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Also-Rallo, Eva
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, Barcelona, Spain Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Le Heron, Anita
论文数: 0 引用数: 0
h-index: 0
机构:
Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Alias, Laura
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, Barcelona, Spain Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Denis, Cecile
论文数: 0 引用数: 0
h-index: 0
机构:
Inst Stem Cell Therapy & Explorat Monogen Dis, Evry, France Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Girard, Mathilde
论文数: 0 引用数: 0
h-index: 0
机构:
Inst Stem Cell Therapy & Explorat Monogen Dis, Evry, France Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Yee, Jiing-Kuan
论文数: 0 引用数: 0
h-index: 0
机构:
City Hope Natl Med Ctr, Beckman Res Inst, Dept Virol, Duarte, CA 91010 USA Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Tizzano, Eduardo F.
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Santa Creu & Sant Pau, Dept Genet, Barcelona, Spain
Hosp Santa Creu & Sant Pau, CIBERER, U 705, Barcelona, Spain Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England

Yanez-Munoz, Rafael J.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England Univ London, Sch Biol Sci, Egham TW20 0EX, Surrey, England
[5]
Pediatric SMA patients with complex spinal anatomy: Implementation and evaluation of a decision-tree algorithm for administration of nusinersen
[J].
Carrera-Garcia, Laura
;
Muchart, Jordi
;
Lazaro, Juan Jose
;
Exposito-Escudero, Jessica
;
Cuadras, Daniel
;
Medina, Julita
;
de Basea, Magda Bosch
;
Colomer, Jaume
;
Jimenez-Mallebrera, Cecilia
;
Ortez, Carlos
;
Natera-de Benito, Daniel
;
Nascimento, Andres
.
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY,
2021, 31
:92-101

Carrera-Garcia, Laura
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain
ISCIII, Ctr Biomed Res Rare Dis CIBERER, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Muchart, Jordi
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Dept Radiol, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Lazaro, Juan Jose
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Dept Anesthesiol, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Exposito-Escudero, Jessica
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain
ISCIII, Ctr Biomed Res Rare Dis CIBERER, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Cuadras, Daniel
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Dept Stat, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Medina, Julita
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Dept Rehabil, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

de Basea, Magda Bosch
论文数: 0 引用数: 0
h-index: 0
机构:
Global Hlth Inst Barcelona ISGlobal, Barcelona, Spain
Univ Pompeu Fabra UPF, Dept Expt & Hlth Sci, Barcelona, Spain
CIBER Epidemiol & Publ Hlth CIBERESP, Madrid, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Colomer, Jaume
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Jimenez-Mallebrera, Cecilia
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Ortez, Carlos
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Natera-de Benito, Daniel
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain

Nascimento, Andres
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain Hosp St Joan de Deu, Neuromuscular Unit, Neuropediat Dept, Inst Recerca Pediat Hosp, Barcelona, Spain
[6]
The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
[J].
d'Ydewalle, Constantin
;
Ramos, Daniel M.
;
Pyles, Noah J.
;
Ng, Shi-Yan
;
Gorz, Mariusz
;
Pilato, Celeste M.
;
Ling, Karen
;
Kong, Lingling
;
Ward, Amanda J.
;
Rubin, Lee L.
;
Rigo, Frank
;
Bennett, C. Frank
;
Sumner, Charlotte J.
.
NEURON,
2017, 93 (01)
:66-79

d'Ydewalle, Constantin
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Ramos, Daniel M.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurosci, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Pyles, Noah J.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Ng, Shi-Yan
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Univ, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA
Harvard Univ, Harvard Stem Cell Inst, Cambridge, MA 02138 USA
Inst Mol & Cell Biol, Neurotherapeut Lab, 61 Biopolis Dr, Singapore 138673, Singapore Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Gorz, Mariusz
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Pilato, Celeste M.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Ling, Karen
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, 2855 Gazelle Court, Carlsbad, CA 92010 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Kong, Lingling
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Ward, Amanda J.
论文数: 0 引用数: 0
h-index: 0
机构:
MIT, Koch Inst Integrat Canc Res, 500 Main St, Cambridge, MA 02142 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Rubin, Lee L.
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Univ, Dept Stem Cell & Regenerat Biol, Cambridge, MA 02138 USA
Harvard Univ, Harvard Stem Cell Inst, Cambridge, MA 02138 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Rigo, Frank
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, 2855 Gazelle Court, Carlsbad, CA 92010 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Bennett, C. Frank
论文数: 0 引用数: 0
h-index: 0
机构:
Ionis Pharmaceut, 2855 Gazelle Court, Carlsbad, CA 92010 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA

Sumner, Charlotte J.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA
Johns Hopkins Univ, Sch Med, Dept Neurosci, 855 North Wolfe St, Baltimore, MD 21205 USA Johns Hopkins Univ, Sch Med, Dept Neurol, 855 North Wolfe St, Baltimore, MD 21205 USA
[7]
Neurofilament as a potential biomarker for spinal muscular atrophy
[J].
Darras, Basil T.
;
Crawford, Thomas O.
;
Finkel, Richard S.
;
Mercuri, Eugenio
;
De Vivo, Darryl C.
;
Oskoui, Maryam
;
Tizzano, Eduardo F.
;
Ryan, Monique M.
;
Muntoni, Francesco
;
Zhao, Guolin
;
Staropoli, John
;
McCampbell, Alexander
;
Petrillo, Marco
;
Stebbins, Christopher
;
Fradette, Stephanie
;
Farwell, Wildon
;
Sumner, Charlotte J.
.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY,
2019, 6 (05)
:932-944

Darras, Basil T.
论文数: 0 引用数: 0
h-index: 0
机构:
Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA
Harvard Med Sch, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 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
Johns Hopkins Univ, Sch Med, Dept Pediat, Baltimore, MD 21205 USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Finkel, Richard S.
论文数: 0 引用数: 0
h-index: 0
机构:
Nemours Childrens Hosp, Div Neurol, Dept Pediat, Orlando, FL USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Mercuri, Eugenio
论文数: 0 引用数: 0
h-index: 0
机构:
Catholic Univ, Dept Paediat Neurol, Rome, Italy Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

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 Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

论文数: 引用数:
h-index:
机构:

Tizzano, Eduardo F.
论文数: 0 引用数: 0
h-index: 0
机构:
Hosp Valle De Hebron, Dept Clin & Mol Genet, Barcelona, Spain
Hosp Valle De Hebron, Rare Dis Unit, Barcelona, Spain
Ctr Invest Biomed Red Enfermedades Rares CIBERER, Barcelona, Spain Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Ryan, Monique M.
论文数: 0 引用数: 0
h-index: 0
机构:
Royal Childrens Hosp, Murdoch Childrens Res Inst, Melbourne, Vic, Australia
Univ Melbourne, Melbourne, Vic, Australia Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Muntoni, Francesco
论文数: 0 引用数: 0
h-index: 0
机构:
UCL, Dubowitz Neuromuscular Ctr, London, England
NIHR Great Ormond St Hosp, Biomed Res Ctr, London, England Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Zhao, Guolin
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Staropoli, John
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

McCampbell, Alexander
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Petrillo, Marco
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Stebbins, Christopher
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Fradette, Stephanie
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Farwell, Wildon
论文数: 0 引用数: 0
h-index: 0
机构:
Biogen, Cambridge, MA USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA

Sumner, Charlotte J.
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 Neurosci, Baltimore, MD 21205 USA Boston Childrens Hosp, Dept Neurol, 330 Longwood Ave,Fegan,11th Floor, Boston, MA 02115 USA
[8]
Biochemical and clinical biomarkers in adult SMA 3-4 patients treated with nusinersen for 22 months
[J].
De Wel, Bram
;
De Schaepdryver, Maxim
;
Poesen, Koen
;
Claeys, Kristl G.
.
ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY,
2022, 9 (08)
:1241-1251

De Wel, Bram
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium
Katholieke Univ Leuven, Leuven Brain Inst LBI, Dept Neurosci, Lab Muscle Dis & Neuropathies, Leuven, Belgium Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium

De Schaepdryver, Maxim
论文数: 0 引用数: 0
h-index: 0
机构:
Katholieke Univ Leuven, Leuven Brain Inst LBI, Dept Neurosci, Lab Mol Neurobiomarker Res, Leuven, Belgium Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium

Poesen, Koen
论文数: 0 引用数: 0
h-index: 0
机构:
Katholieke Univ Leuven, Leuven Brain Inst LBI, Dept Neurosci, Lab Mol Neurobiomarker Res, Leuven, Belgium
Univ Hosp Leuven, Lab Med, Leuven, Belgium Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium

Claeys, Kristl G.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium
Katholieke Univ Leuven, Leuven Brain Inst LBI, Dept Neurosci, Lab Muscle Dis & Neuropathies, Leuven, Belgium Univ Hosp Leuven, Dept Neurol, Campus Gasthuisberg,Herestr 49, B-3000 Leuven, Belgium
[9]
Neuronal-Derived EV Biomarkers Track Cognitive Decline in Alzheimer's Disease
[J].
Eren, Erden
;
Leoutsakos, Jeannie-Marie
;
Troncoso, Juan
;
Lyketsos, Constantine G.
;
Oh, Esther S.
;
Kapogiannis, Dimitrios
.
CELLS,
2022, 11 (03)

Eren, Erden
论文数: 0 引用数: 0
h-index: 0
机构:
NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA

Leoutsakos, Jeannie-Marie
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Sch Med, Dept Psychiat & Behav Sci, Baltimore, MD 21205 USA
Johns Hopkins Sch Med, Div Geriatr Med & Gerontol, Dept Med, Baltimore, MD 21287 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA

Troncoso, Juan
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Sch Med, Dept Neurol, Baltimore, MD 21224 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA

Lyketsos, Constantine G.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Sch Med, Dept Psychiat & Behav Sci, Baltimore, MD 21205 USA
Johns Hopkins Sch Med, Div Geriatr Med & Gerontol, Dept Med, Baltimore, MD 21287 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA

Oh, Esther S.
论文数: 0 引用数: 0
h-index: 0
机构:
Johns Hopkins Sch Med, Div Geriatr Med & Gerontol, Dept Med, Baltimore, MD 21287 USA
Johns Hopkins Sch Med, Richman Family Precis Med Ctr Excellence Alzheime, Baltimore, MD 21224 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA

Kapogiannis, Dimitrios
论文数: 0 引用数: 0
h-index: 0
机构:
NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA
Johns Hopkins Sch Med, Dept Neurol, Baltimore, MD 21224 USA NIA, Intramural Res Program, Lab Clin Invest, Baltimore, MD 21224 USA
[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