共 8 条
[1]
A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy
[J].
Barrett, Doreen
;
Bilic, Sanela
;
Chyung, Yung
;
Cote, Shaun M.
;
Iarrobino, Ryan
;
Kacena, Katherine
;
Kalra, Ashish
;
Long, Kimberly
;
Nomikos, George
;
Place, Amy
;
Still, James Gordon
;
Vrishabhendra, Leela
.
ADVANCES IN THERAPY,
2021, 38 (06)
:3203-3222

Barrett, Doreen
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Bilic, Sanela
论文数: 0 引用数: 0
h-index: 0
机构:
Vanadro LLC, Urbandale, IA USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Chyung, Yung
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Cote, Shaun M.
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Iarrobino, Ryan
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Kacena, Katherine
论文数: 0 引用数: 0
h-index: 0
机构:
Katherine Kacena Consulting, Natick, MA USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Kalra, Ashish
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Long, Kimberly
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Nomikos, George
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Place, Amy
论文数: 0 引用数: 0
h-index: 0
机构:
Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Still, James Gordon
论文数: 0 引用数: 0
h-index: 0
机构:
Clin Dev Solut LLC, Raleigh, NC USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA

Vrishabhendra, Leela
论文数: 0 引用数: 0
h-index: 0
机构:
Medpace Clin Pharmacol, Cincinnati, OH USA Scholar Rock Inc, 301 Binney St,3rd Floor, Cambridge, MA 02142 USA
[2]
Newborn Screening for SMA - Can a Wait-and-See Strategy be Responsibly Justified in Patients With Four SMN2 Copies?
[J].
Blaschek, Astrid
;
Koelbel, Heike
;
Schwartz, Oliver
;
Koehler, Cornelia
;
Glaeser, Dieter
;
Eggermann, Katja
;
Hannibal, Iris
;
Schara-Schmidt, Ulrike
;
Mueller-Felber, Wolfgang
;
Vill, Katharina
.
JOURNAL OF NEUROMUSCULAR DISEASES,
2022, 9 (05)
:597-605

Blaschek, Astrid
论文数: 0 引用数: 0
h-index: 0
机构:
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Koelbel, Heike
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Essen Gesamthsch, Dept Pediat Neurol, Dev Neurol & Social Pediat, Essen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Schwartz, Oliver
论文数: 0 引用数: 0
h-index: 0
机构:
Munster Univ Hosp, Dept Pediat Neurol, Munster, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Koehler, Cornelia
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Bochum, Dept Pediat Neurol, Bochum, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Glaeser, Dieter
论文数: 0 引用数: 0
h-index: 0
机构:
MVZ Genetikum GmbH, Ctr Human Genet, Neu Ulm, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Eggermann, Katja
论文数: 0 引用数: 0
h-index: 0
机构:
Rhein Westfal TH Aachen, Med Fac, Inst Human Genet, Aachen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Hannibal, Iris
论文数: 0 引用数: 0
h-index: 0
机构:
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Schara-Schmidt, Ulrike
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Essen Gesamthsch, Dept Pediat Neurol, Dev Neurol & Social Pediat, Essen, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Mueller-Felber, Wolfgang
论文数: 0 引用数: 0
h-index: 0
机构:
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany

Vill, Katharina
论文数: 0 引用数: 0
h-index: 0
机构:
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, LMU Ctr Children Med Complex, Munich, Germany Ludwig Maximilians Univ Munchen, Dr von Hauner Childrens Hosp, LMU Hosp, Dept Pediat Neurol & Dev Med M, Munich, Germany
[3]
Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme
[J].
D'Silva, Arlene M.
;
Kariyawasam, Didu S. T.
;
Best, Stephanie
;
Wiley, Veronica
;
Farrar, Michelle A.
.
DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY,
2022, 64 (05)
:625-632

D'Silva, Arlene M.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Kariyawasam, Didu S. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Best, Stephanie
论文数: 0 引用数: 0
h-index: 0
机构:
Macquarie Univ, Australian Inst Hlth Innovat, Sydney, NSW, Australia
Murdoch Childrens Res Inst, Australian Genom, Melbourne, Vic, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Wiley, Veronica
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Westmead, NSW Newborn Screening Programme, Westmead, NSW, Australia
Univ Sydney, Fac Med & Hlth, Sydney Med Sch, Discipline Child & Adolescent Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Farrar, Michelle A.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Univ New South Wales Med & Hlth, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
[4]
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
[5]
Presymptomatic spinal muscular atrophy: a cautionary approach to the proposed new terminology
[J].
Farrar, Michelle A.
;
Kiernan, Matthew C.
;
Kariyawasam, Didu S.
.
BRAIN,
2023, 146 (09)
:E65-E66

Farrar, Michelle A.
论文数: 0 引用数: 0
h-index: 0
机构:
UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2031, Australia
Sydney Childrens Hosp Network, Level 8,Bright Alliance Bldg,Avoca St, Randwick, NSW 2031, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia

Kiernan, Matthew C.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Sydney, Brain & Mind Ctr, Sydney, NSW 2050, Australia
Royal Prince Alfred Hosp, Dept Neurol, Sydney, NSW 2050, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia

Kariyawasam, Didu S.
论文数: 0 引用数: 0
h-index: 0
机构:
UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2031, Australia UNSW, Discipline Paediat & Child Hlth, UNSW Med & Hlth, Sydney, NSW 2052, Australia
[6]
Newborn screening for spinal muscular atrophy in Australia: a non-randomised cohort study
[J].
Kariyawasam, Didu S.
;
D'Silva, Arlene M.
;
Sampaio, Hugo
;
Briggs, Nancy
;
Herbert, Karen
;
Wiley, Veronica
;
Farrar, Michelle A.
.
LANCET CHILD & ADOLESCENT HEALTH,
2023, 7 (03)
:159-170

Kariyawasam, Didu S.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW 2035, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

D'Silva, Arlene M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Sampaio, Hugo
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Briggs, Nancy
论文数: 0 引用数: 0
h-index: 0
机构:
Univ New South Wales, Mark Wainwright Analyt Ctr, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Herbert, Karen
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Physiotherapy, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Wiley, Veronica
论文数: 0 引用数: 0
h-index: 0
机构:
Childrens Hosp Westmead, New South Wales Newborn Screening Program, Westmead, NSW, Australia
Univ Sydney, Fac Med & Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia

Farrar, Michelle A.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Sch Clin Med Med & Hlth, Discipline Paediat & Child Hlth, Sydney, NSW, Australia Sydney Childrens Hosp Network, Dept Neurol, Sydney, NSW, Australia
[7]
Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy
[J].
Kariyawasam, Didu S. T.
;
D'Silva, Arlene
;
Lin, Cindy
;
Ryan, Monique M.
;
Farrar, Michelle A.
.
FRONTIERS IN NEUROLOGY,
2019, 10

Kariyawasam, Didu S. T.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia

D'Silva, Arlene
论文数: 0 引用数: 0
h-index: 0
机构:
Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia

Lin, Cindy
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Sydney, Brain & Mind Ctr, Dept Neurophysiol, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia

Ryan, Monique M.
论文数: 0 引用数: 0
h-index: 0
机构:
Univ Melbourne, Royal Childrens Hosp, Dept Neurol, Murdoch Childrens Res Inst, Melbourne, Vic, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia

Farrar, Michelle A.
论文数: 0 引用数: 0
h-index: 0
机构:
Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia
Univ New South Wales, Univ New South Wales Med, Sch Womens & Childrens Hlth, Sydney, NSW, Australia Sydney Childrens Hosp, Dept Neurol, Sydney, NSW, Australia
[8]
Challenges and opportunities in spinal muscular atrophy therapeutics
[J].
Yeo, Crystal J. J.
;
Tizzano, Eduardo F.
;
Darras, Basil
.
LANCET NEUROLOGY,
2024, 23 (02)
:205-218

Yeo, Crystal J. J.
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA
Northwestern Univ, Feinberg Sch Med, Dept Neurol, Chicago, IL USA
Univ Aberdeen, Sch Med Med Sci & Nutr, Aberdeen, Scotland
Nanyang Technol Univ, Lee Kong Chian Sch Med, Singapore, Singapore
Agcy Sci Technol & Res, Singapore, Singapore
Tan Tock Seng, Natl Neurosci Inst, Singapore, Singapore
Singapore Gen Hosp, Singapore, Singapore
Duke NUS Med Sch, Singapore, Singapore Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA

Tizzano, Eduardo F.
论文数: 0 引用数: 0
h-index: 0
机构:
Vall dHebron Univ Hosp, Dept Clin & Mol Genet, Barcelona, Spain
Vall dHebron Res Inst, Genet Med, Barcelona, Spain Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA

Darras, Basil
论文数: 0 引用数: 0
h-index: 0
机构:
Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA Harvard Med Sch, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA